WO2025046421A1

WO2025046421A1 - Methods of using factor b inhibitors

Info

Publication number: WO2025046421A1
Application number: PCT/IB2024/058205
Authority: WO
Inventors: Eder JÖRG; Thomas Holbro; Rajesh Singh Karan; Bernd C. Kieseier; Anna Svenja Schubart Wellensiek
Original assignee: Novartis Ag
Priority date: 2023-08-25
Filing date: 2024-08-23
Publication date: 2025-03-06
Also published as: TW202508580A

Abstract

Described herein are methods of treating gMG (generalized Myasthenia gravis) with the Factor B inhibitor iptacopan or a pharmaceutically acceptable salt or hydrate thereof.

Description

PAT059532 FF METHODS OF USING FACTOR B INHIBITORS FIELD The disclosure relates to methods of treating complement driven diseases, in particular, Myasthenia Gravis (MG), and in particular generalized Myasthenia Gravis (gMG), with the Factor B inhibitor iptacopan or a pharmaceutically acceptable salt or hydrate thereof. BACKGROUND Myasthenia gravis (MG) is a rare autoimmune disorder that affects the postsynaptic membrane of the neuromuscular junction (NMJ). It is caused by autoantibodies against different components of NMJ. The main manifestation of the disease is fluctuating muscle weakness that is typically milder in the morning as a result of overnight inactivity, which enables the replenishment of acetylcholine levels in pre-synaptic motor nerve terminals. However, the symptoms worsen during the course of the day due to repeated muscle activity (Jayam Trouth et al. Autoimmune Dis 2012874680; Gilhus, N Engl J Med, 2016, 2570-81). Infrequently, respiratory muscle weakness can occur, leading to a life-threatening condition known as myasthenic crisis. The Myasthenia Gravis Foundation of America (MGFA) clinical classification divides MG into five main classes and several subclasses. This classification is designed to identify different subgroups of patients who have distinct severity of disease, indicating different prognoses or responses to therapy. Generalized Myasthenia Gravis (gMG) corresponds to MGFA classes II-V (Jayam Trouth et al.2012). Generalized MG manifests in approximately 85% of all MG patients and affects multiple muscle groups throughout the body. Moreover, approximately eighty percent of MG patients have detectable antibodies against the acetylcholine receptor (AChR+), which is the targeted study population for this study. MG has an annual incidence of 8 to 10 cases per 1 million with a prevalence rate of 150 to 250 cases per 1 million, and is thus the most common disease that affects the NMJ (Gilhus 2016). MG primarily occurs at two peaks of incidence. The first early-onset peak affects younger adults, aged between 30 to 50 years. Women are most commonly affected before the age of 40, with a female : male ratio of 3:1. The second peak occurs later in life, with women and men equally affected during their fifth decade. However, after the age of 50, men are more often affected, with PAT059532 FF a male : female ratio of 3:2. Furthermore, in men, the incidence increases steadily with age and reaches the highest rates between 60 and 89 years (Dresser et al., J Clin Med, 2021, 10(11)). Even though there are available therapies for MG, e.g., gMG, most of those therapies work via non-specific suppression of the autoimmune response like corticosteroids (classified as immune suppressive therapies (IST) or non-steroidal immunosuppressive therapies (NSIST)). Moreover, their use comes with disadvantages including risks of infections, malignancies and a broad spectrum of systemic side effects which can negatively impact patient quality of life and disease burden (Bacci et al., BMC Neurol 2019, 335). This is especially true for patients that have other medical comorbidities such as diabetes or hypertension. Also, most NSISTs have a delayed onset of action, with an estimated 10% of patients remaining refractory and others intolerant of conventional IST (Mantegazza et al., Curr Opin Neurol 2018, 517-25). These limitations have more recently strongly encouraged the development of MG treatments that now include various drug classes such as complement inhibitors, neonatal crystallizable fragment receptor (FcRn) receptor blockers, and direct and indirect B cell inhibitors. However, therapies like FcRn receptor blockers do not have durable efficacy as symptoms return toward the end of the treatment cycle (Howard et al.2021). Thus, an unmet need still exists for the MG population, as many of these newer therapies (i.e., eculizumab, ravulizumab, efgartigimod) require repeated administration via intravenous or subcutaneous injection, which can be burdensome for patients. SUMMARY The disclosure relates to methods of treating Myasthenia Gravis (MG), in particular generalized Myasthenia Gravis (gMG), with iptacopan or a pharmaceutically acceptable salt or hydrate thereof. Iptacopan is also known as LNP023. In particular, the disclosure provides a method of treating generalized Myasthenia Gravis (gMG) in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject, e.g., patient, iptacopan or a pharmaceutically acceptable salt or hydrate thereof. The disclosure further provides a method of reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. The disclosure further provides a method of reducing a Quantitative Myasthenia Gravis (QMG) PAT059532 FF total score in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In particular, the disclosure provides iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in a treatment of generalized Myasthenia Gravis (gMG) in a subject, e.g., a patient, in need thereof. The disclosure further provides iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof. The disclosure further provides iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. In particular, the disclosure provides a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in treating generalized Myasthenia Gravis (gMG) in a subject, e.g., a patient, in need thereof. The disclosure further provides a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof. The disclosure further provides a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. In particular, the disclosure provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for treating generalized Myasthenia Gravis (gMG) in a subject, e.g., a patient, in need thereof. The disclosure further provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof. The disclosure further provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. In an embodiment, iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. In another embodiment, iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, from about 100 mg to about 200 mg, at a dose of about 50 mg, about 75 PAT059532 FF mg, about 100 mg, about 150 mg, or about 200 mg, each dose administered twice daily (b.i.d.), e.g., about every 12 hours, to thereby treat the subject, e.g., patient (wherein the dosing amount refers to the anhydrous iptacopan free base). In an embodiment of the methods and uses provided herein, the subject has anti- acetylcholine receptor antibody-positive (AChR+) generalized Myasthenia Gravis (gMG). In further embodiments, the method or uses further comprises administering to the subject an immunosuppressant, a corticosteroid, a non-steroidal immunosuppressive therapy (NSIST), and/or a cholinesterase inhibitor. In an embodiment, the methods or uses provided herein comprises one or more of the following: a. achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b. achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c. achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d. achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e. achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Non-limiting embodiments of the present disclosure are described in the following embodiments: Embodiment 1: A method of treating Myasthenia Gravis (MG), in particular generalized Myasthenia Gravis (gMG), in a subject in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. PAT059532 FF Embodiment 2: The method according to embodiment 1, wherein the subject has anti- acetylcholine receptor antibody-positive (AChR+) gMG. Embodiment 3: The method according to any one of embodiments 1 to 2, wherein the method further comprises achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 4: The method according to embodiment 3, wherein the reduction in a MG- ADL total score is by at least 2 points, in particular the reduction in a MG-ADL total score is by at least 2 points at 6 months compared to at baseline. Embodiment 5: The method according to embodiment 4, wherein the reduction in a MG- ADL total score is by at least 3 points, in particular the reduction in a MG-ADL total score is by at least 3 points at 6 months compared to at baseline. Embodiment 6: The method according to any one of embodiments 1 to 5, wherein the method further comprises achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 7: The method according to embodiment 6, wherein the reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. Embodiment 8: The method according to embodiment 7, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. PAT059532 FF Embodiment 9: The method according to any one of the preceding embodiments, wherein the method comprises one or more of the following: a) achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b) achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c) achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d) achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e) achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 10: A method of reducing a Myasthenia Gravis Activity of Daily Living (MG- ADL) total score in a subject in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. Embodiment 11: The method according to embodiment 10, wherein the method achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, e.g., at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 12: The method according to embodiment 11, wherein the method achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, in particular the method achieves a reduction in a MG-ADL total score by at least 2 points at 6 months compared to at baseline. PAT059532 FF Embodiment 13: The method according to embodiment 12, wherein the method achieves a reduction in a MG-ADL total score by at least 3 points compared to at baseline, in particular the method achieves a reduction in a MG-ADL total score by at least 3 points at 6 months compared to at baseline. Embodiment 14: A method of reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. Embodiment 15: The method according to embodiment 14, wherein the method achieves a reduction in a QMG total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 16: The method according to embodiment 15, wherein the method achieves a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the method achieves a reduction in a QMG total score by at least 4 points at 6 months compared to at baseline. Embodiment 17: The method according to embodiment 16, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. Embodiment 18: The method according to any one of embodiments 1 to 17, wherein the method comprises administering to the subject iptacopan hydrochloride. Embodiment 19: The method according to embodiment 18, wherein the method comprises administering to the subject iptacopan hydrochloride monohydrate. Embodiment 20: The method according to any one of embodiments 1 to 19, wherein the method comprises administering to the subject iptacopan at a dose of from about 50 mg to PAT059532 FF about 500 mg, e.g., from about 50 mg to about 250 mg, or from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). Embodiment 21: The method according to any one of embodiments 1 to 20, wherein the method comprises administering to the subject iptacopan at a dose of about 50 mg, or about 75 mg, or about 100 mg, or about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). Embodiment 22: The method according to any one of embodiments 1 to 21, wherein the dose is about 50 mg twice daily. Embodiment 23: The method according to any one of embodiments 1 to 21, wherein the dose is about 100 mg twice daily. Embodiment 24: The method according to any one of embodiments 1 to 21, wherein the dose is about 200 mg twice daily. Embodiment 25: The method according to any one of embodiments 1 to 24, wherein iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. Embodiment 26: The method according to any one of embodiments 1 to 25, wherein the method further comprises administering to the subject an immunosuppressant. Embodiment 27: The method according to embodiment 26, wherein the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. Embodiment 28: The method according to embodiment 26 or embodiment 27, wherein the subject has been administered the immunosuppressant for at least six months. Embodiment 29: The method according to any one of embodiments 1 to 28, wherein the method further comprises administering to the subject a corticosteroid. PAT059532 FF Embodiment 30: The method according to embodiment 29, wherein the subject has been administered a corticosteroid for at least four weeks. Embodiment 31: The method according to any one of embodiments 1 to 30, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. Embodiment 32: The method according to any one of embodiments 1 to 31, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis and Streptococcus pneumoniae. Embodiment 33: The method according to any one of embodiments 1 to 32, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Haemophilus influenzae. Embodiment 34: The method according to any one of embodiments 1 to 33, wherein the subject is not optimally controlled for greater than or equal to 6 months on one non-steroidal immunosuppressive therapies (NSIST). Embodiment 35: The method according to any one of embodiments 1 to 33, wherein the subject is not optimally controlled for greater than or equal to 6 months on two or more non- steroidal immunosuppressive therapies (NSIST). Embodiment 36: The method according to embodiment 34 or embodiment 35, wherein the NSIST is azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide. PAT059532 FF Embodiment 37: The method according to any one of embodiments 1 to 36, wherein the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved for gMG. Embodiment 38: The method according to any one of embodiments 1 to 37, wherein the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non-steroidal immunosuppressive therapies (NSISTs). Embodiment 39: The method according to any one of embodiments 1 to 38, wherein the method further comprises administering to the subject azathioprine. Embodiment 40: The method according to embodiment 39, wherein the method further comprises administering to the subject azathioprine for at least 6 months. Embodiment 41: The method according to any one of embodiments 1 to 40, wherein the method further comprises administering to the subject a cholinesterase inhibitor. Embodiment 42: The method according to embodiment 41, wherein the method further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. Embodiment 43: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in a treatment of Myasthenia Gravis (MG), in particular generalized Myasthenia Gravis (gMG), in a subject in need thereof. Embodiment 44: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 43, wherein gMG is anti-acetylcholine receptor antibody-positive (AChR+) gMG. PAT059532 FF Embodiment 45: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 44, wherein the use further comprises achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 46: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 45, wherein the reduction in a MG-ADL total score is by at least 2 points compared to at baseline, in particular the reduction in a MG-ADL total score is by at least 2 points at 6 months compared to at baseline. Embodiment 47: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 46, wherein the reduction in a MG-ADL total score is by at least 3 points compared to at baseline, in particular the reduction in a MG-ADL total score is by at least 3 points at 6 months compared to at baseline. Embodiment 48: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 47, wherein the use further comprises achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 49: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 48, wherein the reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. Embodiment 50: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 49, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. PAT059532 FF Embodiment 51: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 50, wherein the use comprises one or more of the following: a) achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b) achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c) achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d) achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e) achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 52: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject in need thereof. Embodiment 53: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 52, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points, e.g., at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 54: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 53, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, in particular the use achieves a reduction in a MG- ADL total score by at least 2 points at 6 months compared to at baseline. PAT059532 FF Embodiment 55: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 54, wherein the use achieves a reduction in a MG-ADL total score by at least 3 points compared to at baseline, in particular the use achieves a reduction in a MG- ADL total score by at least 3 points at 6 months compared to at baseline. Embodiment 56: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject in need thereof. Embodiment 57: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 56, wherein the use achieves a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 58: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 57, wherein the use achieves a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the use achieves a reduction in a QMG total score by at least 4 points at 6 months compared to at baseline. Embodiment 59: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 58, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. Embodiment 60: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 59, wherein the use comprises administering to the subject iptacopan hydrochloride. Embodiment 61: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 60, wherein the use comprises administering to the subject iptacopan hydrochloride monohydrate. PAT059532 FF Embodiment 62: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 61, wherein the use comprises administering to the subject iptacopan at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, or from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). Embodiment 63: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 62, wherein the use comprises administering to the subject iptacopan at a dose of about 50 mg, or about 75 mg, or about 100 mg, or about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). Embodiment 64: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 63, wherein the dose is about 50 mg twice daily. Embodiment 65: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 63, wherein the dose is about 100 mg twice daily. Embodiment 66: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 63, wherein the dose is about 200 mg twice daily. Embodiment 67: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 66, wherein iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. Embodiment 68: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 67, wherein the use further comprises administering to the subject an immunosuppressant. Embodiment 69: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 68, wherein the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. PAT059532 FF Embodiment 70: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 68 or embodiment 69, wherein the subject has been administered the immunosuppressant for at least six months. Embodiment 71: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 70, wherein the use further comprises administering to the subject a corticosteroid. Embodiment 72: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 71, wherein the subject has been administered a corticosteroid for at least four weeks. Embodiment 73: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 72, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. Embodiment 74: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 73, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis and Streptococcus pneumoniae. Embodiment 75: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 74, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Haemophilus influenzae. Embodiment 76: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 75, wherein the subject is not optimally controlled PAT059532 FF for greater than or equal to 6 months on one non-steroidal immunosuppressive therapies (NSIST). Embodiment 77: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 75, wherein the subject is not optimally controlled for greater than or equal to 6 months on two or more non-steroidal immunosuppressive therapies (NSIST). Embodiment 78: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 76 or embodiment 77, wherein the NSIST is azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide. Embodiment 79: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 78, wherein the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved from gMG. Embodiment 80: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 79, wherein the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non-steroidal immunosuppressive therapies (NSISTs). Embodiment 81: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 80, wherein the use further comprises administering to the subject azathioprine. Embodiment 82: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 81, wherein the use further comprises administering to the subject azathioprine for at least 6 months. PAT059532 FF Embodiment 83: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of embodiments 43 to 82, wherein the method further comprises administering to the subject a cholinesterase inhibitor. Embodiment 84: Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to embodiment 83, wherein the method further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. Embodiment 85: A pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in treating Myasthenia Gravis (MG), in particular generalized Myasthenia Gravis (gMG), in a subject in need thereof. Embodiment 86: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 85, wherein gMG is anti-acetylcholine receptor antibody-positive (AChR+) gMG. Embodiment 87: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 86, wherein the use further comprises achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG- ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 88: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 87, wherein the reduction in a MG-ADL total score is by at least 2 points compared to at baseline, in particular the reduction in a MG- ADL total score is by at least 2 points at 6 months compared to at baseline. PAT059532 FF Embodiment 89: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 88, wherein the reduction in a MG-ADL total score is by at least 3 points compared to at baseline, in particular the reduction in a MG- ADL total score is by at least 3 points at 6 months compared to at baseline. Embodiment 90: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 89, wherein the use further comprises achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 91: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 90, wherein the reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. Embodiment 92: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 91, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. Embodiment 93: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of preceding embodiments 85 to 92, wherein the use comprises one or more of the following: PAT059532 FF a) achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b) achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c) achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d) achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e) achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 94: A pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject in need thereof. Embodiment 95: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 94, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points, e.g., at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 96: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 95, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, in particular the use achieves a reduction in a MG-ADL total score by at least 2 points at 6 months compared to at baseline. PAT059532 FF Embodiment 97: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 96, wherein the use achieves a reduction in a MG-ADL total score by at least 3 points compared to at baseline, in particular the use achieves a reduction in a MG-ADL total score by at least 3 points at 6 months compared to at baseline. Embodiment 98: A pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject in need thereof. Embodiment 99: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 98, wherein the use achieves a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. Embodiment 100: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 99, wherein the use achieves a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the use achieves a reduction in a QMG total score by at least 4 points at 6 months compared to at baseline. Embodiment 101: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 100, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. Embodiment 102: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically PAT059532 FF acceptable carrier for use according to any one of embodiments 85 to 101, wherein the use comprises administering to the subject iptacopan hydrochloride. Embodiment 103: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 102, wherein the use comprises administering to the subject iptacopan hydrochloride monohydrate. Embodiment 104: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 103, wherein the use comprises administering to the subject iptacopan at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, or from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). Embodiment 105: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 104, wherein the use comprises administering to the subject iptacopan at a dose of about 50 mg, or about 75 mg, or about 100 mg, or about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). Embodiment 106: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 105, wherein the dose is about 50 mg twice daily. Embodiment 107: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 105, wherein the dose is about 100 mg twice daily. PAT059532 FF Embodiment 108: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 105, wherein the dose is about 200 mg twice daily. Embodiment 109: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 108, wherein iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. Embodiment 110: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 109, wherein the use further comprises administering to the subject an immunosuppressant. Embodiment 111: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 110, wherein the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. Embodiment 112: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 110 or embodiment 111, wherein the subject has been administered the immunosuppressant for at least six months. Embodiment 113: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 112, wherein the use further comprises administering to the subject a corticosteroid. PAT059532 FF Embodiment 114: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 113, wherein the subject has been administered a corticosteroid for at least four weeks. Embodiment 115: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 114, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. Embodiment 116: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 115, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis and Streptococcus pneumoniae. Embodiment 117: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 116, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Haemophilus influenzae. Embodiment 118: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 117, wherein the subject is not optimally controlled for greater than or equal to 6 months on one non-steroidal immunosuppressive therapies (NSIST). PAT059532 FF Embodiment 119: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 117, wherein the subject is not optimally controlled for greater than or equal to 6 months on two or more non-steroidal immunosuppressive therapies (NSIST). Embodiment 120: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 118 or embodiment 119, wherein the NSIST is azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide. Embodiment 121: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 120, wherein the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved for gMG. Embodiment 122: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 121, wherein the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non-steroidal immunosuppressive therapies (NSISTs). Embodiment 123: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 122, wherein the use further comprises administering to the subject azathioprine. PAT059532 FF Embodiment 124: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 123, wherein the use further comprises administering to the subject azathioprine for at least 6 months. Embodiment 125: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of embodiments 85 to 124, wherein the use further comprises administering to the subject a cholinesterase inhibitor. Embodiment 126: The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to embodiment 125, wherein the use further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. Embodiment 127: Use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for treating Myasthenia Gravis (MG), in particular generalized Myasthenia Gravis (gMG), in a subject in need thereof. BRIEF DESCRIPTION OF THE DRAWINGS Figure 1. LNP023 protects from passive-transfer induced experimental autoimmune myasthenia gravis (PT-EAMG). Anti-AChR (mAb35) was injected at time 0, and animals were monitored twice daily for A) clinical symptoms and B) body weight. C)There was a trend for reduced disease duration, D) Maximal disease scores were significantly reduced by LNP023. E) Combined muscle action potential (cMAP) was measured at 36h post disease induction; no significant difference after LNP023 treatment. F) disease onset was significantly reduced by LNP023. Mean and standard error of the mean are shown. In C-E, each point represents an individual animal. Statistical analysis was performed using GraphPad Prism version 10.1.2. using Kruskal Wallis test followed by Dunn’s multiple comparison test. For cMAP, Brown-Forsythe and Welch ANOVA test was used followed by Dunnett’s T3 multiple comparison test. For disease PAT059532 FF onset, Log rank (Mantel-Cox) test was used to compare mAb35 vehicle-treated group with mAb 35 LNP023-treated group * p<0.05. DETAILED DESCRIPTION MG is considered a classic example of antibody-mediated autoimmune disease. It can also be viewed as an example of a class II hypersensitivity reaction, as IgG autoantibodies react with intra or extracellular antigens, leading to end-organ damage. Most patients with MG have autoantibodies against acetylcholine receptors, and a minority are seropositive for antibodies directed to muscle specific tyrosine kinase (MuSK) or low density lipoprotein receptor-related protein 4 (LRP4). These antibodies also provide the basis for defining disease subgroups and help delineate phenotypic variants (Dresser et al.2021). AChR antibodies are predominantly of the IgG1 and IgG3 subclasses. IgG2 and IgG4 subclasses are also identified, but in fewer cases. The pathogenic mechanisms and functional spectrum of AChR antibodies are varied, but overall, they impair receptor function by either binding, blocking, or modulating its activity. There are three mechanisms by which AChR autoantibodies act to reduce synaptic transmission at the NMJ: 1. Blocking the binding of acetylcholine (ACh) to AChRs; 2. Accelerating the internalization and degradation of AChRs that are cross-linked by autoantibodies; 3. Complement activation. The predominant mechanism is the binding of the antibody and activation of the complement cascade, leading to the formation of the membrane attack complex (MAC), which causes damage of the postsynaptic membrane and destruction of synaptic folds that contain AChRs and associated proteins, including voltage-gated sodium channels (Morgan et al., Clin Exp Immunol, 2006, 294-302 and Howard et al., Exp Opin Investig Drugs 2017, 483-93). Iptacopan is a first-in-class, oral, low molecular weight (LMW) inhibitor of Factor B (FB) (Schubart et al.2019, Small-molecule factor B inhibitor for the treatment of complement-mediated diseases. Proc Natl Acad Sci U S A p. 7926-7931), a key protease of the AP (Merle et al. 2015, Complement System Part I -Molecular Mechanisms of Activation and Regulation. Front Immunol p. 262). Inhibition of FB prevents amplification of all pathways as well as AP-induced assembly of C3- and C5-convertases. At the same time, iptacopan has only limited effect on classical- PAT059532 FF pathway induced activation of the terminal pathway. Iptacopan inhibits FB in the context of the C3 convertase and thereby blocks AP-dependent C3 activation and the amplification of CP- and LP-dependent C5 activation. Iptacopan does not, however, block the generation of MAC initiated by CP and LP. This is important, since it means that in immunized individuals, MAC-dependent killing of Neisseria species through activation of CP will be maintained. The terms “iptacopan” and “LNP023” are used herein interchangeably. Iptacopan, IUPAC name: (4-((2S,4S)-(4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2- yl))benzoic acid), belongs to the class of Factor B inhibitors of the complement pathway and acts by inhibiting or suppressing the amplification of the complement system caused by C3 activation irrespective of the initial mechanism of activation. Iptacopan hydrochloride is chemically designated as 4-((2S,4S)-(4-ethoxy-1-((5-methoxy- 7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl))benzoic acid hydrochloride of the following Formula (I):

Formula I. Iptacopan hydrochloride and methods for its preparation are disclosed in International Application No. PCT/US2014/046515 (WO 2015/009616, see Example 26d), which is incorporated herein by reference in its entirety. In an embodiment, iptacopan hydrochloride of Formula (I) is provided as a crystalline solid. In a preferred embodiment, iptacopan hydrochloride of Formula (I) is provided as a crystalline monohydrate form, which is hereinafter also referred to as “Form HB”. The Form H_B of iptacopan hydrochloride monohydrate is as shown in the formula below: PAT059532 FF

(2S,4S)-2-(4-Carboxyphenyl)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4- yl)methyl]piperidin-1-ium chloride―water (1/1) In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the PXRD (powder X-ray diffraction) peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (9.2 ± 0.2)°, and (19.1 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha_1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, and (19.1 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by having a powder X-ray diffractogram comprising one or more peaks at 2-Theta angles selected from (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (19.1 ± 0.2)° and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 °C to 30 °C with a Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (12.2 ± 0.2)°, (19.1 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha_1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (12.2 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. PAT059532 FF In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (16.6 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°,and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (16.6 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha_1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (16.6 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the crystalline form of LNP023 hydrochloride (Form HB) can be characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha_1,2 radiation having a wavelength of 0.15419 nm. PAT059532 FF In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, (24.0 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, (22.2 ± 0.2)°, (24.0 ± 0.2)°, and (24.6 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, (22.2 ± 0.2)°, (24.0 ± 0.2)°, (24.6 ± 0.2)°and (28.0 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha_1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by the PXRD peaks identified at 2-Theta angles of (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (12.2 ± 0.2)°, (19.1 ± 0.2)°, and (24.6 ± 0.2)°, and at least one more peak selected from the group consisting of (10.0 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, (22.2 ± 0.2)°, (24.0 ± 0.2)°, and (28.0 ± 0.2)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha_1,2 radiation having a wavelength of 0.15419 nm. In another embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the PXRD peaks identified at 2-Theta angles of: (4.6 ± 0.1)°, (9.2 ± 0.1)°, and (19.1 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, and (19.1 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (19.1 ± 0.1)°, and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (12.2 ± 0.1)°, (19.1 ± 0.1)°, and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (12.2 ± 0.1)°, (19.1 ± 0.1)°, (21.3 ± 0.1)°, and (24.6 ± 0.1)°; or PAT059532 FF (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (12.2 ± 0.1)°, (12.6 ± 0.1)°, (16.6 ± 0.1)°, (19.1 ± 0.1)°, (21.3 ± 0.1)°,and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (10.0 ± 0.1)°, (12.2 ± 0.1)°, (12.6 ± 0.1)°, (16.6 ± 0.1)°, (19.1 ± 0.1)°, (21.3 ± 0.1)°,and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (10.0 ± 0.1)°, (12.2 ± 0.1)°, (12.6 ± 0.1)°, (16.6 ± 0.1)°, (19.1 ± 0.1)°, (21.3 ± 0.1)°, and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (10.0 ± 0.1)°, (12.2 ± 0.1)°, (12.6 ± 0.1)°, (15.3 ± 0.1)°, (16.6 ± 0.1)°, (19.1 ± 0.1)°, (21.3 ± 0.1)°, and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (10.0 ± 0.1)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°; or (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, and (24.6 ± 0.2)°; or (4.6 ± 0.2)°, (6.8 ± 0.2)°, (9.2 ± 0.2)°, (10.0 ± 0.2)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.2)°, (16.6 ± 0.2)°, (17.2 ± 0.2)°, (19.1 ± 0.2)°, (20.7 ± 0.2)°, (21.3 ± 0.2)°, (24.0 ± 0.2)°, and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (10.0 ± 0.1)°, (12.2 ± 0.1)°, (12.6 ± 0.1)°, (15.3 ± 0.1)°, (16.6 ± 0.1)°, (17.2 ± 0.1)°, (19.1 ± 0.1)°, (20.7 ± 0.1)°, (21.3 ± 0.1)°, (22.2 ± 0.1)°, (24.0 ± 0.1)°, and (24.6 ± 0.1)°; or (4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (10.0 ± 0.1)°, (12.2 ± 0.2)°, (12.6 ± 0.2)°, (15.3 ± 0.1)°, (16.6 ± 0.1)°, (17.2 ± 0.1)°, (19.1 ± 0.1)°, (20.7 ± 0.1)°, (21.3 ± 0.1)°, (22.2 ± 0.1)°, (24.0 ± 0.1)°, (24.6 ± 0.1)°and (28.0 ± 0.1)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by the PXRD peaks identified at 2-Theta angles of (4(4.6 ± 0.1)°, (6.8 ± 0.1)°, (9.2 ± 0.1)°, (12.2 ± 0.1)°, (19.1 ± 0.1)°, and (24.6 ± 0.1)°, and at least one more peak selected from the group consisting of (10.0 ± 0.1)°, (12.6 ± 0.1)°, (15.3 ± 0.1)°, (16.6 ± 0.1)°, (17.2 ± 0.1)°, (20.7 ± 0.1)°, (21.3 ± 0.1)°, (22.2 ± 0.1)°, (24.0 ± 0.1)°, and (28.0 ± 0.1)°, when measured at a temperature in the range of from 20 to 30 °C with Cu-Kalpha1,2 radiation having a wavelength of 0.15419 nm. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by having a Fourier transform infrared spectrum comprising peaks at wavenumbers of (3452 ± 2) cm- PAT059532 FF ¹, (2875 ± 2) cm^-1, (1692 ± 2) cm^-1, (1439 ± 2) cm^-1 and (1243 ± 2) cm^-1, when measured at a temperature in the range of from 20 °C to 30 °C with a diamond ATR cell. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by having an FTIR spectrum comprising peaks at wavenumbers of (3452± 4) cm^-1, (2875 ± 4) cm^-1, (1692 ± 4) cm^-1, (1439 ± 4) cm^-1, (1243 ± 4) cm^-1 and (767± 4) cm^-1, when measured at a temperature in the range of from 20 to 30 °C with a diamond ATR cell. In an embodiment, iptacopan hydrochloride monohydrate (Form H_B) is characterized by having an FTIR spectrum comprising peaks at wavenumbers of (3452± 4) cm^-1, (2875 ± 4) cm^-1, (2732 ± 4) cm^-1, (1692 ± 4) cm^-1, (1439 ± 4) cm^-1, (1243 ± 4) cm^-1, and (767± 4) cm^-1, when measured at a temperature in the range of from 20 to 30 °C with a diamond ATR cell. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by having a DSC (differential scanning calorimetry) curve showing a broad endothermic event which ends at about 170°C, followed by exothermic decomposition at about 200°C, when measured at a heating rate of 10 K/min. In an embodiment, iptacopan hydrochloride monohydrate (Form HB) is characterized by having a differential scanning calorimetry curve comprising an endothermic event in the range of from 35 °C to 170 °C, when measured at a heating rate of 10 K/min. Iptacopan hydrochloride monohydrate (Form H_B) is characterized by having a thermogravimetric analysis curve showing a mass loss at about 220°C, such as at a temperature of from 200 °C to 220°C, due to loss of water and residual solvents of not more than 4.5 weight%, , e.g., of not more than 4.3w-%, e.g., of not more than 4.0w-%, e.g., of not more than 3.8 w-%, for example of not more than 3.4w-%, based on the weight of the crystalline form, when heated from 30 °C to 300 °C at a rate of 20 K/min. Iptacopan hydrochloride monohydrate (Form H_B) is characterized by showing a mass change of not more than 4.5 weight%, e.g., of not more than 4.0 w-%, e.g., of not more than 3.0 w-%, e.g., of not more than 2.0 w-%, for example of not more than 1.8 w%, 1.6 w-%, 1.5 w-% or 1.4w-%, based on the weight of the crystalline form at 0% relative humidity, when measured with dynamic vapor sorption at a relative humidity in the range of from 0% to 95% and a temperature of (25 ± 1.0) °C. Iptacopan hydrochloride monohydrate (Form HB) advantageously has a crystal habit which is essentially equant or columnar in shape. PAT059532 FF Iptacopan hydrochloride monohydrate (Form H_B) typically has an aspect ratio of from about 0.4 to about 0.7 such as from about 0.45 to about 0.6. Iptacopan hydrochloride monohydrate (Form HB) advantageously has a particle size distribution X90 of from about 30 μm to about 150 μm, such as from about 35 μm to about 130 μm, for example, from about 40 μm to about 105 μm. Iptacopan hydrochloride monohydrate (Form HB) advantageously has a particle size distribution X₅₀ of from about 5 μm to about 100 μm, such as from about 10 μm to about 70 μm, for example, from about 15 μm to about 55 μm. Iptacopan hydrochloride monohydrate (Form H_B) advantageously has a particle size distribution X10 of from about 0.1 μm to about 50 μm, such as from about 1 μm to about 30 μm, for example, from about 2 μm to about 20 μm. Iptacopan hydrochloride monohydrate (Form HB) generally has a consolidated (15 kPa) bulk density of from about 0.40 g/ml to about 0.70 g/ml, such as from about 0.50 g/ml to about 0.65 g/ml, for example, from about 0.55 g/ml to about 0.60 g/ml. Iptacopan hydrochloride monohydrate (Form H_B) and methods for its preparation are disclosed in U.S.S.N. 63/026,637 and U.S.S.N. 63/052,699, published in WO 2021/234544, each of which is incorporated herein by reference in its entirety. Iptacopan hydrochloride monochloride (Form HB) is advantageously used as the investigational drug for the study of the present disclosure (see examples below). Described herein is a method of treating generalized Myasthenia Gravis (gMG) in a subject in need thereof comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. Accordingly, described herein are methods of treating gMG in a patient in need thereof, the method comprising orally administering, e.g., in capsule form, to the patient a twice daily dose, e.g., about every 12 hours, of iptacopan or a pharmaceutically acceptable salt or hydrate thereof (wherein the dosing amount refers to the anhydrous iptacopan free base). Also described herein is a method of reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof, and/or reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. Described herein is iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in a treatment of generalized Myasthenia Gravis (gMG) in a subject, e.g., a patient, in need thereof. PAT059532 FF Also described herein is iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof, and/or in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. Also described herein a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in treating generalized Myasthenia Gravis (gMG) in a subject, e.g., a patient, in need thereof. Also described herein a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof, and/or in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. The disclosure further provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. Also described herein are methods of selecting the target patient population, methods of monitoring treatment of the target patient population, and methods of assessing safety and efficacy of treatment of the target patient population. The details of the disclosure are set forth in the accompanying description below. Although methods and materials similar or equivalent to those described herein can be used in the practice or testing of the present disclosure, illustrative methods and materials are now described. Other features, objects, and advantages of the disclosure will be apparent from the description and from the claims. In the specification and the claims, the singular forms also include the plural unless the context clearly dictates otherwise. Unless defined otherwise, all technical and scientific terms used herein have the same meaning as commonly understood by one of ordinary skill in the art to which this disclosure belongs. All patents and publications cited in this specification are incorporated herein by reference in their entireties. Definitions Unless specific definitions are provided, the nomenclature used in connection with, and the procedures and techniques of, analytical chemistry, synthetic organic chemistry, and medicinal PAT059532 FF and pharmaceutical chemistry described herein are those well known and commonly used in the art. Standard techniques may be used for chemical synthesis, and chemical analysis. Certain such techniques and procedures may be found for example in “Remington's Pharmaceutical Sciences,” Mack Publishing Co., Easton, Pa., 21st edition, 2005, which is hereby incorporated by reference for any purpose. Where permitted, all patents, applications, published applications and other publications and other data referred to throughout in the disclosure are incorporated by reference herein in their entirety. Unless otherwise indicated, the following terms have the following meanings: As used herein, “about” means within ±10% of a value in addition to including the value or parameter per se. As used herein, the term “about” includes and describes the value or parameter per se. For example, “about x” includes and describes “x” per se. As used herein, the term “about” when used in association with a measurement, or used to modify a value, a unit, a constant, or a range of values, refers to variations of ±1-10%, e.g., ±10%, in addition to including the value or parameter per se. In some embodiments, the term “about” when used in association with a measurement, or used to modify a value, a unit, a constant, or a range of values, refers to variations of ±1, ±2, ±3, ±4, ±5, ±6, ±7, ±8, ±9, or ±10%. As used herein, “administering” or “administration” means providing a pharmaceutical agent to an individual, and includes, but is not limited to, administering by a medical professional and self-administering. Administration of a pharmaceutical agent to an individual can be continuous, chronic, short or intermittent. As used herein, the term “acquire” or “acquiring” as the terms are used herein, refer to obtaining possession of a physical entity (e.g., a sample, e.g., a blood sample or a blood plasma sample), or a value, e.g., a numerical value, by “directly acquiring” or “indirectly acquiring” the physical entity or value. “Directly acquiring” means performing a process (e.g., an analytical method) to obtain the physical entity or value. “Indirectly acquiring” refers to receiving the physical entity or value from another party or source (e.g., a third party laboratory that directly acquired the physical entity or value). Directly acquiring a value includes performing a process that includes a physical change in a sample or another substance, e.g., performing an analytical process which includes a physical change in a substance, e.g., a sample, performing an analytical method, e.g., a method as described herein, e.g., by sample analysis of bodily fluid, such as blood by, e.g., mass spectroscopy, e.g., LC-MS, e.g., LC-MS/MS methods. PAT059532 FF As used herein, “baseline” refers to a time prior to treatment in relation to a characteristic of a subject or a patient. As used herein, “dose” means a specified quantity of a pharmaceutical agent provided in a single administration, or in a specified time period. In certain embodiments, a dose can be administered in capsules. As used herein, the dosing amount refers to the anhydrous iptacopan free base. As used herein, “individual”, “patient”, “participant”, or “subject” means a human selected for treatment or therapy. As used herein, the term “adult” means an individual who is equal to or more than 18 years of age. As used herein, the term “adolescent” means an individual who is between 12 to 17 years of age. As used herein, “pharmaceutically acceptable salts” means physiologically and pharmaceutically acceptable salts of iptacopan, i.e., salts that retain the desired biological activity of iptacopan and do not impart undesired toxicological effects thereto. The term “pharmaceutically acceptable salt” or “salt” includes a salt prepared from pharmaceutically acceptable non-toxic acids or bases, including inorganic or organic acids and bases. “Pharmaceutically acceptable salts” of iptacopan may be prepared by methods well-known in the art. For a review of pharmaceutically acceptable salts, see Stahl and Wermuth, Handbook of Pharmaceutical Salts: Properties, Selection and Use (Wiley-VCH, Weinheim, Germany, 2002). Iptacopan hydrochloride and methods for its preparation are disclosed in WO 2015/009616 (see Example 26d), which is incorporated herein by reference in its entirety. As used herein, “hydrate” means a crystalline form containing one or more water molecules in a three-dimensional periodic arrangement. It can include non-stoichiometric hydrates or stoichiometric hydrates, such as hemihydrates, monohydrates, dihydrates and trihydrates. As used herein, the term “treat”, “treating” or “treatment” means decrease, suppress, attenuate, diminish, arrest, or stabilize the development or progression of a disorder or disease, e.g., gMG. The term “treat”, “treating” or “treatment” includes therapeutic treatments, prophylactic treatments and applications in which one reduces the risk that a subject will develop a disorder, e.g., MG, e,g, gMG, or other risk factor. Treatment does not require the complete curing of a disorder and encompasses the reduction of the symptoms or underlying risk factors. As used herein, the terms “treat”, “treatment” and “treating” refer to the reduction or amelioration of the progression or severity of MG, e.g., gMG, or the amelioration of one or more symptoms, suitably PAT059532 FF of one or more discernible symptoms of MG, e.g., gMG. In specific embodiments, the terms “treat”, “treatment” and “treating” refer to the amelioration of at least one measurable physical parameter of MG, e.g., gMG (such as achieve or at least partially achieve a desired effect (e.g., a partial reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, a partial reduction in a Quantitative Myasthenia Gravis (QMG) total score, a partial reduction in a Myasthenia Gravis Composite (MGC) total score, a partial reduction in a MG-QOL15r survey score, a partial increase in a EQ-5D-5L survey score on EQ VAS scale).As used herein, the term “Myasthenia Gravis Activity of Daily Living (MG-ADL) total score” refers to a clinical assessment tool used to measure the degree of physical impairment and disability of patients with Myasthenia Gravis. The score is based on eight items, including the severity of weakness in ocular, bulbar, respiratory and limb muscles, where each item is graded from 0 (normal) to 3 (severe impairment), leading to a range of scores from 0 to 24. A higher score indicates greater impairment. As used herein, the term “Quantitative Myasthenia Gravis (QMG) total score” refers to a clinical tool used to quantify the severity of muscle weakness in patients with Myasthenia Gravis. The total QMG score is calculated based on the assessment of 13 items, which include various muscle groups and functions such as ocular, bulbar, respiratory, and limb muscles. Each item is rated from 0 (no symptoms) to 3 (severe weakness), thus the total QMG score ranges from 0 to 39, with higher scores signifying more severe muscle weakness. It helps to monitor the progression of the disease and evaluate the effectiveness of the treatment. As used herein, the term “Myasthenia Gravis Composite (MGC) total score” refers to a clinical assessment tool used to evaluate the clinical severity of muscle weakness in individuals with Myasthenia Gravis. The MGC score is derived from a total of 10 items which assess ocular, bulbar, respiratory, and gross motor functions. Each item is scored based on the severity of the symptom, with higher scores indicating greater severity or impairment. The combined total score can range from 0, indicating no clinical weakness, to 50, indicating severe muscle weakness. This is used to track disease progression and response to treatment in patients with MG. As used herein, the term “MG-QOL15r survey” refers to a disease-specific questionnaire used to evaluate the impact of Myasthenia Gravis on a patient's quality of life. The survey consists of fifteen questions covering different aspects of life that may be affected by the disease, including physical, emotional, and social functions. Each question is scored from 0 (not at all) to 4 (very much). Patients answer these questions based on their experiences over the past two weeks, and PAT059532 FF the overall scores range from 0 to 60, with higher scores denoting worse quality of life. The MG- QOL15r survey, therefore, provides a patient-reported outcome measure useful for both clinical practice and research. As used herein, the term “EQ-5D-5L survey” refers to a standardized instrument for measuring generic health status, developed by the EuroQol Group. It provides a simple, generic measure of health for clinical and economic appraisal, and can be used in a wide range of health conditions and treatments. The survey consists of two parts: the EQ-5D descriptive system and the EQ visual analogue scale (EQ-VAS). The descriptive system comprises of five dimensions including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. The EQ-VAS records the respondent's self-rated health on a vertical scale, where the endpoints are labelled 'Best imaginable health state' and 'Worst imaginable health state'. The scores from these two parts provide an overall health profile and measure quality of life for the individual. They are especially used for measuring the effectiveness of various healthcare treatments and interventions. As used herein, the term “equant” refers to crystals which are equidimensional, such as cubes or spheres. As used herein, the term “columnar” refers to elongated, prismatic crystals having greater width and thickness than needles. Such crystal habit definitions are consistent with those usually used in the art, e.g., see “Polymorphism in the Pharmaceutical Industry” edited by Rolf Hilfiker (Wiley-VCH, 2006); Chapter 7, Light Microscopy (Gary Nichols). Unless otherwise specified, conventional definitions of terms control and conventional stable atom valences are presumed and achieved in all formulas and groups. The articles “a” and “an” are used in this disclosure to refer to one or more than one (e.g., to at least one) of the grammatical object of the article. By way of example, “an element” means one element or more than one element. Unless otherwise required by context, singular terms used herein shall include pluralities and plural terms shall include the singular. Uses and Methods In one aspect, provided herein is a method of treating Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG), in a subject in need thereof comprising administering to PAT059532 FF the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In another aspect, provided herein is a method of reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In a further aspect, provided herein is a method of reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In an embodiment, provided herein is a method of treating Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG), in a subject, e.g., a patient, in need thereof, or a method of reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof, or a method of reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof, the method comprising administering, e.g., orally, to the subject, e.g., patient, iptacopan or a pharmaceutically acceptable salt or hydrate thereof at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, from about 100 mg to about 200 mg, at a dose of about 50 mg, about 75 mg, about 100 mg, about 150 mg, or about 200 mg, each dose administered twice daily (b.i.d.), e.g., about every 12 hours (wherein the dosing amount refers to the anhydrous iptacopan free base), to thereby treat the subject, e.g., patient. In another aspect, the disclosure provides iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in the treatment of Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG), in a subject, e.g., a patient, in need thereof. In another aspect, the disclosure provides iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof. In another aspect, the disclosure provides iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. In an embodiment, the treatment or use comprises administering, e.g., orally, to the subject, e.g., patient, iptacopan or a pharmaceutically acceptable salt or hydrate thereof at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, from about 100 mg to about 200 mg, at a dose of about 50 mg, about 75 mg, about 100 mg, about 150 mg, or about 200 mg, each dose to be administered twice daily (b.i.d.), e.g., about every 12 hours (wherein the dosing amount refers to the anhydrous iptacopan free base). PAT059532 FF In another aspect, the disclosure provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof in the manufacture of a medicament for the treatment of Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG), in a subject, e.g., a patient, in need thereof. In another aspect, the disclosure provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof. In another aspect, the disclosure provides use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. In an embodiment, the treatment or use comprises orally administering to the subject, e.g., patient, iptacopan or a pharmaceutically acceptable salt or hydrate thereof at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, from about 100 mg to about 200 mg, at a dose of about 50 mg, about 75 mg, about 100 mg, about 150 mg, or about 200 mg, each dose to be administered twice daily (b.i.d.), e.g., about every 12 hours (wherein the dosing amount refers to the anhydrous iptacopan free base), to thereby treat the subject, e.g., patient. In an embodiment, the subject, e.g., patient, has or is diagnosed as having gMG. In another aspect, the disclosure provides a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in treating Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG), in a subject, e.g., patient, in need thereof. In another aspect, the disclosure provides a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject, e.g., a patient, in need thereof. In another aspect, the disclosure provides a pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject, e.g., a patient, in need thereof. In an embodiment, the pharmaceutical composition is to be administered orally, at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, from about 100 mg to about 200 mg, at a dose of about 50 mg, about 75 mg, about 100 mg, about 150 mg, or about 200 mg of iptacopan or a pharmaceutically acceptable salt or hydrate thereof, each dose to be administered twice daily (b.i.d.), e.g., about PAT059532 FF every 12 hours (wherein the dosing amount refers to the anhydrous iptacopan free base), to thereby treat the subject, e.g., patient. The following embodiments apply to any of the foregoing aspects provided herein and may be combined in any order. In an embodiment, the method or use comprises administering to the subject iptacopan hydrochloride. In another embodiment, the method comprises administering to the subject iptacopan hydrochloride monohydrate (Form H_B). In an embodiment, the pharmaceutical composition comprises iptacopan hydrochloride, and at least one pharmaceutically acceptable carrier for use in treating Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG). In another embodiment, the pharmaceutical composition comprises iptacopan hydrochloride monohydrate (Form HB), and at least one pharmaceutically acceptable carrier for use in treating Myasthenia Gravis, in particular generalized Myasthenia Gravis (gMG). In yet another embodiment, the method or use comprises administering to the subject iptacopan at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). In still another embodiment, the method or use comprises administering to the subject iptacopan at a dose of about 50 mg, about 75 mg, about 100 mg, about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). In an embodiment, the dose is about 50 mg twice daily. In another embodiment, the dose is about 100 mg twice daily. In yet another embodiment, the dose is about 200 mg twice daily. In still another embodiment, iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. In an embodiment, the subject has Myasthenia Gravis Foundation of America (MGFA) Class II-IV MG. In an embodiment, the subject has anti-acetylcholine receptor antibody-positive (AChR+) gMG. In an embodiment, the anti-acetylcholine receptor (AChR) antibody is at least one of IgG1, IgG2, IgG3 and IgG4 subclasses. In an embodiment, the anti-acetylcholine receptor (AChR) antibody is at least one of IgG1 and IgG3 subclasses. In an embodiment, the subject has refractory gMG. PAT059532 FF In an embodiment, the subject has refractory anti-acetylcholine receptor antibody-positive (AChR+) gMG. In an embodiment, the subject, e.g., patient, is an adult. In some embodiments, the adult subject is 18 years of age or older. In one embodiment, the subject, e.g., patient, is an adolescent. In some embodiments, the adolescent subject ranges from 12 to 18 years of age. In another embodiment, the method further comprises administering to the subject an immunosuppressant. In yet another embodiment, the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. In still another embodiment, the subject has been administered the immunosuppressant for at least six months. In an embodiment, the method further comprises administering to the subject a corticosteroid. In another embodiment, the subject has been administered a corticosteroid for at least four weeks. In an embodiment, the method further comprises administering to the subject a cholinesterase inhibitor. In another embodiment, the method further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. In an embodiment, the method further comprises administering to the subject at least quarterly plasmapheresis, plasma exchange, or intravenous immunoglobulin. In an embodiment, the method further comprises administering to the subject at least one of an FcRN antagonist, i.e., efgartigimod, rozanolixizumab. In an embodiment, the method further comprises administering to the subject a complement inhibitor, e.g., eculizumab, ravulizumab, zilucoplan. In an embodiment, the method further comprises administering to the subject a B cell inhibitor, e.g., inebilizumab. In an embodiment, the subject, e.g., patient, has been vaccinated prior to treatment with iptacopan or a pharmaceutically acceptable salt or hydrate thereof, e.g., prior to administering iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In an embodiment, the subject, e.g., patient, has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. In an embodiment, the subject, e.g., patient, has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate PAT059532 FF thereof against Neisseria meningitidis and Streptococcus pneumoniae. In an embodiment, the subject, e.g., patient, has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. In an embodiment, the subject, e.g., patient, has been vaccinated against Neisseria meningitidis (types A, C, Y, W-135 and B) prior to administering iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In an embodiment, the subject, e.g., patient, has been vaccinated against Streptococcus pneumoniae (Pneumovax-23) prior to administering iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In an embodiment, the subject, e.g., patient, has been vaccinated against Haemophilus influenzae prior to administering iptacopan or a pharmaceutically acceptable salt or hydrate thereof. In another embodiment, the subject is not optimally controlled for greater than or equal to 6 months on one non-steroidal immunosuppressive therapy (NSIST). In yet another embodiment, the subject is not optimally controlled for greater than or equal to 6 months on two or more non- steroidal immunosuppressive therapies (NSIST). In still another embodiment, the NSIST is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. In an embodiment, the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved from gMG. In another embodiment, the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non-steroidal immunosuppressive therapies (NSISTs). In an embodiment, the method further comprises administering to the subject azathioprine. In another embodiment, the method further comprises administering to the subject azathioprine for at least 6 months. In an embodiment, the subject has a MG-ADL total score of at least 6 (>50% of the MG- ADL score due to non-ocular symptoms) at baseline. In an embodiment, the method or use further comprises achieving a reduction in a MG-ADL total score, e.g., by at least 2 points, at least PAT059532 FF 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. In an embodiment, the method or use further comprises achieving a reduction in a QMG total score, e.g., by at least 3 points, at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. In an embodiment, the method or use further comprises achieving a reduction in Myasthenia Gravis Composite (MGC) total score by at least 3 points, e.g., at least 4 points, at least 5 points, or least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. In an embodiment, the methods or uses provided herein comprises one or more of the following: a. achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b. achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c. achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d. achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e. achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. In a specific embodiment, the methods or uses provided herein comprises achieving a reduction in a MG-ADL total score is by at least 2 points, in particular the reduction in a MG- ADL total score is by at least 2 points at 6 months compared to at baseline. In a more specific embodiment, the methods or uses provided herein comprises achieving a reduction in a MG- ADL total score is by at least 3 points, in particular the reduction in a MG-ADL total score is by at least 3 points at 6 months compared to at baseline. PAT059532 FF In a specific embodiment, the methods or uses provided herein comprises achieving a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. In a more specific embodiment, the methods or uses provided herein comprises achieving a reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. In a specific embodiment, the methods or uses provided herein comprises (i) achieving a reduction in a MG-ADL total score is by at least 2 points, in particular the reduction in a MG- ADL total score is by at least 2 points at 6 months compared to at baseline, and (ii) achieving a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. In a more specific embodiment, the methods or uses provided herein comprises (i) achieving a reduction in a MG-ADL total score is by at least 3 points, in particular the reduction in a MG- ADL total score is by at least 3 points at 6 months compared to at baseline, and (ii) achieving a reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline.. In an embodiment, the method further comprises achieving a reduction in corticosteroid dose. In an embodiment, the corticosteroid is an oral corticosteroid. EXAMPLES The disclosure is further illustrated by the following examples, which are not to be construed as limiting this disclosure in scope or spirit to the specific procedures herein described. It is to be understood that the examples are provided to illustrate certain embodiments and that no limitation to the scope of the disclosure is intended thereby. It is to be further understood that resort may be had to various other embodiments, modifications, and equivalents thereof which may suggest themselves to those skilled in the art without departing from the spirit of the present disclosure and/or scope of the appended claims. List of abbreviations PAT059532 FF AE Adverse Event b.i.d. bis in die/twice a day CP Complement Pathway CRF Case Report/Record Form (paper or electronic) EDC Electronic Data Capture eGFR Estimated glomerular filtration rate i.v. intravenous IN Investigator Notification mg milligram(s) mL milliliter(s) p.o. oral(ly) PD Pharmacodynamic(s) PK Pharmacokinetic(s) QD Once a day SAE Serious Adverse Event ULN upper limit of normal Glossary of terms Additional Medicinal products that may be used during the clinical trial as described treatment in the protocol, but not as an investigational medicinal product (e.g., any background therapy) Assessment A procedure used to generate data required by the study Biologic Samples A biological specimen including, for example, blood (plasma, serum), saliva, tissue, urine, stool, etc. taken from a study participant Cohort A specific group of participants fulfilling certain criteria and generally treated at the same time Control drug A study drug (active or placebo) used as a comparator to reduce assessment bias, preserve blinding of investigational drug, assess internal study validity, and/or evaluate comparative effects of the investigational drug Dosage Dose of the study treatment given to the participant in a time unit (e.g., 100 mg once a day, 75 mg twice a day) Electronic Data Electronic data capture (EDC) is the electronic acquisition of clinical Capture (EDC) study data using data collection systems, such as Web-based applications, PAT059532 FF interactive voice response systems and clinical laboratory interfaces. EDC includes the use of Electronic Case Report Forms (eCRFs) which are used to capture data transcribed from paper source forms used at the point of care End of the clinical The end of the clinical trial is defined as the last visit of the last participant trial or at a later point in time as defined by the protocol Enrollment Point/time of participant entry into the study at which informed consent must be obtained Estimand A precise description of the treatment effect reflecting the clinical question posed by the trial objective. It summarizes at a population-level what the outcomes would be in the same patients under different treatment conditions being compared. Attributes of an estimand include the population, variable (or endpoint) and treatment of interest, as well as the specification of how the remaining intercurrent events are addressed and a population-level summary for the variable. Healthy volunteer A person with no known significant health problems who volunteers to be a study participant Intercurrent events Events occurring after treatment initiation that affect either the interpretation or the existence of the measurements associated with the clinical question of interest. Investigational The drug whose properties are being tested in the study drug/ treatment Medication number A unique identifier on the label of medication kits Mis-randomized Mis-randomized participants are those who were not qualified for participants randomization and who did not take study treatment, but have been inadvertently randomized into the study Other treatment Treatment that may be needed/allowed during the conduct of the study (i.e. concomitant or rescue therapy) Part A sub-division of a study used to evaluate specific objectives or contain different populations. For example, one study could contain a single dose part and a multiple dose part, or a part in participants with established disease and in those with newly-diagnosed disease Participant A trial participant (can be a healthy volunteer or a patient) Participant number A unique number assigned to each participant upon signing the informed consent. This number is the definitive, unique identifier for the participant and should be used to identify the participant throughout the study for all data collected, sample labels, etc. Period The subdivisions of the trial design (e.g., Screening, Treatment, Follow- up) which are described in the Protocol. Periods define the study phases and will be used in clinical trial database setup and eventually in analysis PAT059532 FF Personal data Participant information collected by the Investigator that is coded for the purpose of the clinical trial. This data includes participant identifier information, study information and biological samples. Premature Point/time when the participant exits from the study prior to the planned participant completion of all study drug administration and/or assessments; at this withdrawal time all study drug administration is discontinued and no further assessments are planned Randomization A unique identifier assigned to each randomized participant number Screen Failure A participant who did not meet one or more criteria that were required for participation in the study Source Source data refers to the initial record, document, or primary location from Data/Document where data comes. The data source can be a database, a dataset, a spreadsheet or even hard-coded data, such as paper or eSource Start of the clinical The start of the clinical trial is defined as the signature of the informed trial consent by the first participant Study treatment Any drug or combination of drugs or intervention administered to the study participants as part of the required study procedures; includes investigational drug(s), control(s) or background therapy Study treatment When the participant permanently stops taking any of the study drug(s) discontinuation prior to the defined study treatment completion date (if any) for any reason; may or may not also be the point/time of study discontinuation Treatment A treatment arm/group defines the dose and regimen or the combination, arm/group and may consist of 1 or more cohorts. Treatment of The treatment of interest and, as appropriate, the alternative treatment to interest which comparison will be made. These might be individual interventions, combinations of interventions administered concurrently, e.g., as add-on to standard of care, or might consist of an overall regimen involving a complex sequence of interventions. This is the treatment of interest used in describing the related clinical question of interest, which might or might not be the same as the study treatment. Variable (or The variable (or endpoint) to be obtained for each participant that is endpoint) required to address the clinical question. The specification of the variable might include whether the participant experiences an intercurrent event. Withdrawal of Withdrawal of consent from the study occurs only when a participant does study consent not want to participate in the study any longer and does not allow any (WoC) further collection of personal data PAT059532 FF Example 1. A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of iptacopan in patients with generalized Myasthenia Gravis (gMG), followed by an open label extension phase Purpose The purpose of this study is to evaluate the efficacy, safety, and tolerability of iptacopan in patients with anti-acetylcholine receptor antibody-positive (AChR+) gMG who are on stable, standard-of-care (SOC) treatment. Iptacopan is administered to the patients as the iptacopan hydrochloride monohydrate (Form HB) as defined herein. Methods for preparation of iptacopan hydrochloride monohydrate (Form HB) are disclosed in Examples 1, 2 and 3 of WO 2021/234544. The study aims to evaluate whether treatment with iptacopan will result in the reduction of the total score in Myasthenia Gravis Activity of Daily Living (MG-ADL) scale as compared to placebo. The MG-ADL is a patient-reported scale that measures MG symptoms and functional status, and is a commonly used scale in gMG studies. This Phase III study will assess the efficacy and safety of iptacopan, compared to placebo, in patients with gMG who are AChR+. This patient population is selected because of iptacopan’s mechanism of action as an inhibitor of the alternative complement pathway, combined with the role of the complement system in the pathophysiology of AChR+ myasthenia gravis, which is further supported by the evidence that anti-complement therapies provide a benefit in gMG. Objectives, Endpoints, and Estimands Table 1 Objectives(s) Endpoint(s) Primary Objectives Endpoint(s) for primary objective(s) ^ To demonstrate efficacy of iptacopan ^ Change from baseline to Month 6 in compared to placebo in patients with Myasthenia Gravis Activity of Daily gMG on stable SOC in reducing the Living (MG-ADL) total score total score of the Myasthenia Gravis Activity of Daily Living (MG-ADL) scale at 6 months (Day 180) of treatment Secondary Objective(s) Endpoint(s) for secondary objective(s) PAT059532 FF Key Secondary Objectives Key Secondary Endpoints ^ To assess whether iptacopan is ^ Change from baseline to Month 6 in superior to placebo in patients with Quantitative MG (QMG) total score gMG ^ Proportion of participants with ≥ 5 o in reducing Quantitative MG points reduction from baseline to (QMG) total score at 6 months of Month 6 of QMG total score without treatment rescue medication and strongly o on the proportion of study confounding prohibited medication o participants achieving a reduction ^ Proportion of participants with ≥ 3 from baseline to Month 6 of QMG points reduction from baseline to total score ≥ 5 points Month 6 of MG-ADL total score o on the proportion of study without rescue medication and participants achieving a reduction strongly confounding prohibited from baseline to Month 6 of MG- medication ADL total score ≥ 3 points ^ Change from baseline to Month 6 in o in reducing MGC total score at Myasthenia Gravis Composite Month 6 of treatment (MGC) total score o in reducing MG-QOL15r survey ^ Change from baseline to Month 6 in score at Month 6 of treatment revised MG Quality of Life Questionnaire (MG-QOL15r) survey score Other Secondary Objectives (Core Other Secondary Endpoints (Core Part) Part) ^ To evaluate the safety and tolerability ^ Incidence of adverse events and of iptacopan compared to placebo in changes in clinical laboratory values, patients with gMG vital signs, and electrocardiograms ^ To evaluate the efficacy of iptacopan ^ Proportion of time patients showed a compared to placebo in patients with reduction from baseline of ≥ 2 points gMG on other efficacy endpoints in MG-ADL score, up to the end of the study ^ Proportion of early MG-ADL responders during treatment (early responders with first MG-ADL improvement from baseline of ≥ 2 points occurring by week 4) ^ Change from baseline to Month 6 in EuroQol-5 Dimensions-5 Level (EQ-5D-5L) Other Secondary Objectives Other Secondary Endpoints (Extension Part) (Extension Part) PAT059532 FF ^ To assess long-term effect of ^ Change from baseline in MG-ADL iptacopan in patients with gMG in total score reducing the total score of the MG- ^ Proportion of participants achieving a ADL during the Extension Part reduction from core part in oral ^ To assess long-term effect of corticosteroids (OCS) dose till the iptacopan in patients with gMG on end of extension part the proportion of study participants ^ Incidence of adverse events and achieving a reduction from Core Part changes in clinical laboratory values, in oral corticosteroids (OCS) dose till vital signs, and electrocardiograms, the end of Extension Part Columbia Suicide Severity Rating ^ To assess long-term safety and tolerability of iptacopan compared to placebo in patients with gMG Primary Estimands The primary clinical question of interest is: What is the effect of iptacopan treatment versus placebo on the change in MG-ADL total score from baseline to 6 months of treatment in patients with gMG who are receiving stable SOC myasthenia gravis treatment, regardless of discontinuation from study treatment for any reason and change in the allowed SOC MG treatment, and as if rescue medication and strongly confounding prohibited medication had not been taken? The justification for the primary estimand is that it will capture both the effect of the study treatment and the effect of allowed SOC MG treatment, mirroring the conditions in clinical practice. The primary estimand is described by the following attributes: ^ Population: Participants between 18-75 years of age, who have a MG-ADL score of at least 6 (≥50% non-ocular) at baseline and are receiving stable SOC myasthenia gravis treatment. For further details about the population, see Inclusion/Exclusion criteria. ^ Endpoint: change from baseline to Month 6 in MG-ADL total score. ^ Treatment of interest: the randomized treatment (iptacopan or placebo) with or without the allowed SOC MG treatment regardless of the change in the allowed SOC MG treatment. Handling of intercurrent events: PAT059532 FF 1. Discontinuation from study treatment for any reason will be handled according to treatment policy strategy, i.e., data after treatment discontinuation will be collected and used for the primary analysis. 2. Change in the allowed SOC MG treatment will be handled according to treatment policy strategy, i.e., data after change of allowed SOC MG treatment will be collected and used for the primary analysis. 3. Intake of rescue medication will be handled irrespective of potential occurrence of other intercurrent events according to hypothetical strategy as if the participant had not taken the rescue medication and would have continued to be treated. Rescue medication has great potential to confound the interpretation of the treatment effect especially given the placebo- controlled setting of the study where participants randomized to the placebo tend to take rescue medication more often than participants randomized in the iptacopan group. Measurements after the rescue medication will be excluded from the analysis and will be imputed via a modelling approach accounting for the rescue medication as potentially indicative of a worsening of condition. 4. Intake of strongly confounding prohibited medication will be handled according to hypothetical strategy irrespective of potential occurrence of other intercurrent events as if the participant had not taken strongly confounding prohibited medication and would have continued to be treated. Prohibited medication has great potential to confound the interpretation of the treatment effect, especially given the placebo-controlled setting of the study where participants randomized to the placebo tend to take other prohibited medication more often than participants randomized in the iptacopan group. Measurements after the intake of strongly confounding prohibited medication will be excluded from the analysis and will be imputed via a modelling approach accounting for the rescue medication as potentially indicative of a worsening of condition. The summary measure: the difference of mean change in MG-ADL total score from baseline to Month 6 between the treatments. Secondary Estimands Change from baseline to Month 6 in QMG total score The clinical question of interest for this secondary estimand is defined similarly as that for primary estimand but instead on change from baseline to Month 6 in QMG total score. The PAT059532 FF population, treatment or interest, summary measure, intercurrent events and their corresponding handling strategies are the same as that outlined above. Proportion of participants with 5 points reduction from baseline to Month 6 of QMG total score without rescue medication and strongly confounding prohibited medication The clinical question of interest for this secondary estimand is: What is the effect of iptacopan treatment versus placebo on the proportion of participants with ≥5 points reduction from baseline to Month 6 of QMG total score without rescue medication and strongly confounding prohibited medication in patients with generalized myasthenia gravis who are receiving stable SOC myasthenia gravis treatment, regardless of discontinuation from study treatment for any reason and change in the allowed SOC MG treatment? The population and treatment of interest are the same as for the primary estimand. Other attributes are listed below. Endpoint: Proportion of participants with 5 points reduction from baseline to Month 6 of QMG total score without rescue medication and strongly confounding prohibited medication. Handling of intercurrent events: 1. Discontinuation from study treatment for any reason will be handled according to treatment policy strategy, i.e., data after treatment discontinuation will be collected and used for the primary analysis. 2. Change in the allowed SOC MG treatment will be handled according to treatment policy strategy, i.e., data after change of allowed SOC MG treatment will be collected and used for the primary analysis. 3. Intake of rescue medication will be handled according to composite strategy irrespective of potential occurrence of other intercurrent events. Intake of rescue medication is considered as treatment failure hence a part of endpoint definition, deemed as non-responder. 4. Intake of strongly confounding prohibited medication will be handled according to composite strategy irrespective of potential occurrence of other intercurrent events. Intake of strongly confounding prohibited medication is considered as treatment failure hence a part of endpoint definition, deemed as non-responder. The summary measure: odds ratio from a logistic regression model adjusted for covariates. Proportion of participants with 3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and strongly confounding prohibited medication PAT059532 FF The clinical question of interest for this secondary estimand is defined similarly above but instead on proportion of participants with 3 points reduction from baseline to Month 6 of MG-ADL total score without rescue medication and strongly confounding prohibited medication. The population, treatment or interest, summary measure, intercurrent events and their corresponding handling strategies are the same as that above. Change from baseline to Month 6 in MGC total score The clinical question of interest for this secondary estimand is defined similarly as that for primary estimand but instead on change from baseline to Month 6 in MGC total score. The population, treatment or interest, summary measure, intercurrent events and their corresponding handling strategies are the same as that above. Change from baseline to Month 6 in MG-QOL15r survey score The clinical question of interest for this secondary estimand is defined similarly as that for primary estimand but instead on change from baseline to Month 6 in MG-QOL15r survey score. The population, treatment or interest, summary measure, intercurrent events and their corresponding handling strategies are the same as that above. Study Design The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan at a dose of 200 mg orally b.i.d or matching placebo, for 6 months (Day 180) while continuing on a stable SOC treatment. The randomization will be stratified based on region. The study consists of a Core Part and an Extension Part. The Core Part consists of 3 periods: Screening Period: A period of up to 8 weeks to assess participants eligibility and to allow the required vaccinations to be administered (vaccinations should be started as early as possible to avoid extension of the screening period). The screening period can be completed in less than 8 weeks, if the participants have already previously received all required vaccinations. Double-Blind Treatment Period: A 6-month randomized, double-blind, placebo-controlled treatment period for the primary efficacy and safety analyses PAT059532 FF Safety Follow-up Period: A period consisting of 2 safety follow-up visits for all participants not continuing treatment in the Extension part or discontinuing early. The Extension Part consists of 2 periods: Open label Extension Period: A 24-month open-label, single arm treatment period in which eligibleparticipants will receive iptacopan treatment Safety Follow-up Period: A period consisting of 2 safety follow-up visits for all participants in the open-label extension CORE PART: Screening Period At screening, participants will be asked to review and sign the informed consent form(s) (ICFs) before performing any study-related assessments. After signing the ICF, inclusion and exclusion criteria will be assessed during the screening visit to verify participants' eligibility for enrollment into the study. Participants must have positive serology testing for AChR+ antibodies as well as a documented history of gMG diagnosis, as supported by at least 1 of the following 3 tests: ^ History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation ^ History of positive edrophonium chloride test ^ Demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors For the purpose of determining eligibility to the study, all participants will need to have blood taken at screening for assessing for the presence of AChR+ antibodies by central laboratory testing. Other screening assessments include a physical examination, vital signs, infection surveillance, demography, medical history, prior/concomitant medications, electrocardiogram (ECG), hematology, clinical chemistry and urinalysis. For participants requiring vaccination per inclusion criteria, the screening period will last a maximum of 8 weeks (-56 days) prior to Baseline (BL), to ensure time for vaccination or re- vaccination. However, while the screening period for these unvaccinated participants may last for up to 8 weeks, to ensure greater accuracy of recordings for eligibility, safety assessments (particularly clinical laboratory safety tests) are required to be performed within 2 weeks (-14 days) prior to BL. For fully vaccinated participants, the screening can be reduced to 2 weeks (-14 days) which includes conduct of all screening assessments prior to BL. PAT059532 FF All efforts should be made to have the final vaccine given at least 2 weeks prior to initiation of iptacopan treatment. If there is no other option that to start iptacopan sooner than 2 weeks post- vaccination, participant must be given prophylactic antibiotic at the start of iptacopan until at least 2 weeks after vaccination. Throughout the protocol, “baseline” refers to the Day 1 visit (pre-dose). Vaccinations must be completed in accordance with the Inclusion criteria defined below. Vaccines should cover as many serotypes as possible (including meningococcal serotypes A, C, Y, W‑135 and B). To minimize participant burden, the use of multivalent vaccines is recommended as locally available and per local guidelines and regulations (e.g., quadrivalent vaccine for N. meningitis, which covers serotypes A, C, Y and W-135 and Pneumovax-23 which covers 23 S. pneumoniae serotypes). For the vaccination type and booster requirements use local guidelines, and locally available vaccines (and refer to the package insert of those, or local guidelines). Participants who are being treated with stable doses of standard of care treatment for gMG at the time of screening visit are required to continue these medications throughout the Double-blind Treatment Period and Open-label Extension Period. Dosage of the SOC medications must not be changed and no new SOC medication may be added or discontinued during the double blind treatment period of the study, unless changes are deemed medically necessary by the Investigator. If study eligibility criteria are not met, the participant should be screen failed. However, in some instances, for participants who fail eligibility during the screening process due to a temporary condition (such as safety laboratory values), re-testing may be permitted within the same screening period on a case-by-case basis to redetermine eligibility. Participants that do not meet the eligibility criteria during the screening period can be re-screened at a later date and randomized into the study if all inclusion and exclusion criteria are met, however this must be discussed on a case-by-case basis. Screen-failed participants undergoing rescreening will be required to sign another ICF and a new participant number will be assigned. Double-blind Treatment Period Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan at a dose of 200 mg orally b.i.d or matching placebo, for 6 months (Day 180) while continuing on a stable SOC treatment. The randomization will be stratified based on region. At the Baseline visit, before randomization, all participants will be reassessed for eligibility based on the study inclusion and exclusion criteria. The MG-ADL scale needs to be performed prior to all other efficacy or quality of life assessments followed by other patient-reported scales. PAT059532 FF Baseline values for vital signs, ECG, hematology, clinical chemistry, biomarkers, myasthenia gravis related assessments and scales as well as the quality of life questionnaires will be completed at this visit. Study treatment will start on the first day of dosing (Day 1) and continue for 6 months (Day 180). Participants will return to the study center for scheduled visits on Days 15, 30, 60, 90, 120, 150 and 180 of the study, and the study efficacy and safety assessments will be performed at these visits. Blood sampling for pharmacokinetics and biomarkers will be collected pre-dose (i.e., prior to administration of study drug) on Day 15, 30, 90, 150 and, EOS visit of the Double-blind Treatment Period. Interactive Response Technology (IRT) contacts, study drug dispensing and accountability will be performed as required at each of the study visits. Dose adjustment of iptacopan is not allowed during the randomized treatment period and further details on dose interruptions or discontinuation of study drug can be found below. Furthermore, the dose of the SOC medication must remain stable and unchanged throughout the treatment period. However, per the Investigator's judgment and definition for deterioration as described below, if there is a deterioration of the participant's MG status or a risk of MG crisis, the participant is permitted to receive rescue therapy. This rescue therapy can either be IVIG or plasmapharesis/plasma exchange as described below and the participant is allowed to continue on the blinded study treatment if it is considered safe per Investigator medical judgement. In such cases, the rescue therapy used for a given participant will be at the discretion of the Investigator. Note: alternative or additional rescue medications as described below are not permitted. Participants receiving prohibited medication must be discontinued from study treatment. The Double-blind Treatment Period will end with completion of the Month 6 (Day 180) visit assessments. All participants will have an EOS visit for the core part either upon completion of the Month 6 (Day 180) visit or if the participant prematurely discontinues the treatment period (i.e., discontinues from double-blind study treatment and is not willing to attend any further subsequent study visits as per schedule). Upon completion of all assessments at the Month 6 visit (Day 180), all eligible participants who have completed the Double-blind treatment period will be offered to continue in the OLE and will receive iptacopan for a period of 24 months as outlined below. PAT059532 FF For participants who permanently discontinue treatment during the Double-blind Treatment Period, efforts should be made to encourage these participants to remain in the study for further monitoring and complete all assessments up to Day 180 (Month 6/EOS visit). Participants who prematurely discontinue double-blind study treatment and who are willing to continue in the trial will have an End of Treatment (EOT) visit at the time of double-blind study medication discontinuation and these participants will not be eligible to enter the open label extension part. These discontinued participants will complete Safety Follow-up visits 7 days and 30 days after the Month 6 EOT/EOS visit. Participants who complete the double-blind treatment period, including those who received rescue therapy and for whom continuation of the study treatment was safely warranted, are eligible to enter the open label extension part of the study and are not required to complete the Safety Follow- up visit. Because of the known risk of complement inhibitor treatment for infections with encapsulated bacteria, most importantly Neisseria meningitidis, all participants will be provided with a Participant Safety Card. Participants will be instructed to be vigilant for any clinical sign of bacterial infections and to contact the investigator or local physician immediately in case of suspicion of infection and start of antibiotic treatment as soon as possible. An interim analysis (IA) will be conducted by an independent team on change from baseline in MG-ADL and QMG at Month 3 data when 33% of patients are enrolled, to provide the opportunity for an early stop of the trials for futility. Recruitment will continue while the IA is ongoing. Prior to the completion of enrollment into the Core Part of the study, a blinded data analysis (i.e., the treatment code will not be broken), will be performed to reassess sample size assumptions for primary endpoint once 70% of patients have been enrolled in the study. Based on this blinded analysis, the number of participants to be enrolled may be increased to a maximum number of 190 randomized participants. Safety Follow-up Period A period consisting of 2 safety follow-up assessment, one 7 days after the last administration of study treatment (i.e., Month 6 EOT/EOS +7 days) and one 30 days after the last administration of study treatment (i.e., EOT/EOS +30 days) for all participants not continuing treatment in the Extension part. This follow-up may be a phone call or a study site visit. The information collected for the Month 6 EOT/EOS +7 visit will be collected on the eCRF. The information collected for PAT059532 FF the Month 6 EOT/EOS +30 visit will be entered in the eCRF. SAE reporting continues during this time period as described below. Documentation of attempts to contact the participant are required to be recorded in the source documentation. Extension Part Open-label Extension Period Participants completing all visits on study treatment through Month 6 (Day 180) of the Double- blind Treatment Period will be eligible to participate in the open label extension treatment period (OLE) with all participants receiving iptacopan 200 mg b.i.d. This will be a blinded transition from the Double-blind Treatment Period of study to OLE. Participants eligible and agreeing participate in the OLE treatment will start treatment upon completion of the Month 6 (Day 180) visit after completing all assessments of the Double-blind Treatment Period. Efficacy and safety assessments performed at this visit will be used for the EOS visit of the Double-blind Treatment Period as well as for the enrollment/Baseline Visit (OLE Day 1). Participants not eligible or not agreeing to continue in the OLE after completing 6 months of double-blind treatment will complete EOS assessments. IRT contacts, study treatments dispensing, and accountability will be performed at each visit. All participants will return for the Safety Follow-up visit 30 days after the Month 24 EOT/EOS visit. Safety Follow-up Period A period consisting of two safety follow-up assessments, one 7 days after the last administration of study treatment (i.e., Month 24 EOT/EOS +7 days) and one 30 days after the last administration of study treatment (i.e., Month 24 EOT/EOS +30 days) for all participants in the Extension part. This follow-up may be a phone call or a study site visit. The information collected for the Month 24 EOT/EOS +7 visit will be collected on the eCRF. The information collected for the Month 24 EOT/EOS +30 visit will be entered in the eCRF. SAE reporting continues during this time period as described below. Documentation of attempts to contact the participant are required to be recorded in the source documentation. Treatment of Interest The randomized treatment (iptacopan 200 mg b.i.d. or matching placebo) with stable standard of care (SOC) treatment Rationale for choice of control drugs or combination drugs and background therapy PAT059532 FF Participants in this clinical trial may continue to receive stable standard of care therapy as assigned by their treating physician. Participants receiving SOC therapy will be required to have been on this therapy for a defined period of time prior to baseline (see inclusion criteria) and remain on the same stable therapy throughout the duration of the randomized period of this clinical trial. Except when rescue medication is permitted, any change in SOC, because of disease worsening or progression, might result in discontinuing the participant from receiving the investigational treatment (iptacopan or placebo). This study allows for inclusion of patients treated with a spectrum of NSISTs (which include azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide), corticosteroids, and acetylcholinesterase inhibitors, which are commonly prescribed for gMG, and are often taken by patients in combination. These treatments are expected to vary from patient to patient based on investigator/treating physician clinical judgment given the clinical context and local SOC practices. A stable dose of preexisting treatment for gMG (e.g., acetylcholinesterase inhibitors, corticosteroids, or NSIST) before baseline and throughout the trial is permitted and required per inclusion criteria. For cholinesterase inhibitors, increases in cholinesterase therapy that are required as a result of intercurrent illness or other medical cause of deterioration are permitted, but dosing should be returned to dosing levels at study entry as soon as feasible and the Sponsor should be notified of the change. The dose of corticosteroids, or NSISTs needs to remain stable throughout the study duration. Prohibited medications are listed in below. Participants who have not previously responded to other complement inhibitors are excluded, as it is unlikely that they would respond to iptacopan given the very similar mechanism of action (see inclusion/exclusion criteria). Patients will be randomized 1:1 to receive either iptacopan 200 mg b.i.d. or matching placebo for the 6-month double-blind treatment period to assess efficacy, safety, and tolerability. For this study, a placebo control on top of stable SOC background therapy (which will be allowed in both treatment arms) has been selected due to the unstable and potentially life-threatening nature of the disease and is considered appropriate to maintain a balance between a robust evaluation of efficacy and safety in this registration study and ensuring that patients are maintained on the SOC so as to prevent decompensation. This risk is further mitigated by not including MGFA class V patients. Therefore, patients randomized to the placebo group are not expected to experience PAT059532 FF significant harm during the course of the 6-month treatment period, even in the absence of other, more recently approved therapies. Furthermore, in case of disease worsening or disease progression, patients may receive rescue treatment, IVIG or PLEX, as per local treatment guidance. At the conclusion of the double-blind treatment period, all patients who have completed the double-blind treatment period will have the option to roll over into the open-label extension where they would receive iptacopan 200 mg b.i.d. Treatment Groups Participants will be assigned at Randomization visit (Day 1) to one of the following 2 treatment arms/groups in a ratio of 1:1 ^ Iptacopan arm: iptacopan orally at 200 mg b.i.d. for 6 months (double-blind) followed by open-label iptacopan at 200 mg b.i.d. for an additional 24 months ^ Placebo arm: placebo orally for 6 months (double-blind) followed by open-label iptacopan at 200 mg b.i.d. for 24 months End of study definition The end of the study is defined as the date of the last visit of the last participant in the study or last scheduled procedure or follow-up for the last participant in the study globally. This study includes a Core part and an Extension part. Study completion for the Core part is defined as when the last participant finishes their End of Study visit and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator or, in the event of an early study termination decision, the date of that decision. Participants who complete the Double-blind Treatment Period may be eligible to enroll in the Extension part. Study completion for the Extension part is defined as when the last participant finishes their End of Study visit and any repeat assessments associated with this visit have been documented and followed-up appropriately by the Investigator or, in the event of an early study termination decision, the date of that decision. Participants who prematurely withdraw from the Double-blind Treatment Period for any reason are not eligible to enroll in the Extension part. All randomized and/or treated participants who, for whatever reason, do not continue into the Extension part should have a safety follow-up visits 7 days and 30 days after completing the end of study treatment visit (Month 6/EOT/EOS). All participants who continue into the open-label PAT059532 FF Extension part and discontinue treatment for any reason or complete all visits should have a safety follow-up visit conducted 7 days and 30 days after completing the Month 24/EOT/EOS visit. This follow up may be a phone call or a study site visit. The information collected will be recorded in the respective eCRF. SAE reporting continues during this time period. Documentation of attempts to contact the participant are required to be recorded in the source documentation. Number of Participants The study will enroll approximately 146 male and female patients, 18-75 years of age diagnosed with gMG, with disease categorized by MGFA as class II to IV, who are treated with a stable regimen of SOC treatment (see inclusion/exclusion criteria for requirements for the SOC treatments), but still presenting with sub-optimally controlled symptoms of gMG (i.e., MG-ADL of score of at least 6 with ≥50% of the MG-ADL score due to non-ocular symptoms) at baseline. The defined trial population will include patients with AChR+ antibodies only. At least 20% of enrolled participants should have been treated with just one NSIST. Inclusion Criteria Participants eligible for inclusion in this study must meet all of the following criteria: 1. Patients with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures 2. Adult patients with generalized Myasthenia Gravis (age 18-75 years) 3. Positive serology testing for AChR+ antibody at screening 4. MGFA Class II-IV gMG and likely not in need of a respirator for the duration of the study, as judged by the Investigator. 5. The confirmation of the diagnosis of gMG should be documented and supported by one of the following three tests: ^ History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation ^ History of positive edrophonium chloride test ^ Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician 6. Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms 7. Participants not optimally controlled for ≥6 months on ^ just one NSIST; or PAT059532 FF ^ two or more NSISTs; or ^ on frequent (at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and NSISTs; or ^ one of the following gMG treatments: ^ a FcRN antagonist approved for gMG ^ rituximab ^ other approved gMG therapies excluding complement inhibitors Note: Non-steroidal immunosuppressive therapies (NSIST) include azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide 8. Participants who are receiving azathioprine, are required to have been on azathioprine for at least 6 months and on a stable dose for at least 2 months prior to baseline 9. Participants who are receiving other immunosuppressive therapies (i.e., mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide), are required to be on these immunosuppressive therapies for at least 6 months and to have been on a stable dose for at least 1 month prior to baseline 10. Participants receiving oral corticosteroids are required to be on a stable dose for at least 4 weeks (i.e., 28 days or longer) prior to baseline 11. Participants receiving a cholinesterase inhibitor, are required be on a stable dose for at least 2 weeks prior to baseline 12. Consistent with all other iptacopan trials, participants will have to be vaccinated against Neisseria meningitidis and Streptococcus pneumoniae. In addition, participants will be vaccinated against Haemophilus influenzae, depending on the local regulations and on the availability of this vaccine in the countries of study conduct. The vaccination will be performed at least 2 weeks prior to first dosing with iptacopan, covering as many serotypes as possible. If iptacopan treatment will start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment must be initiated and administered until 2 weeks post vaccination. Key Exclusion Criteria Participants meeting any of the following criteria are not eligible for inclusion in this study: 1. Prior to baseline have been treated with IVIg/PLEX in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement PAT059532 FF inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV): serologic panel test results indicative of an active (acute or chronic) infection; Active Hepatitis C Virus (HCV): serology positive for HCV-Ab; Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count ≤200 cells/mm3 History of hypersensitivity to any of the study drugs or its excipients or to drugs of similar chemical classes to iptacopan Female participants who are pregnant or lactating, or are intending to become pregnant Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment. Effective contraception methods include: ^ Total abstinence (when this is in line with the preferred and usual lifestyle of the participant. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post- ovulation methods) and withdrawal are not acceptable methods of contraception ^ Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least six weeks before taking investigational drug. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment ^ Male sterilization (at least 6 months prior to screening). For female participants on the study, the vasectomized male partner should be the sole partner for that participant ^ Barrier methods of contraception: condom or occlusive cap (e.g., diaphragm or cervical/vault caps) ^ Use of oral (estrogen and progesterone), injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example, hormone vaginal ring or transdermal hormone contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS). PAT059532 FF ^ In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment ^ Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., age-appropriate history of vasomotor symptoms). Women are considered not of child-bearing potential if they are post-menopausal or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least six weeks prior to enrollment on study. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow-up hormone level assessment is she considered to be not of child-bearing potential ^ If local regulations are more stringent than the contraception methods listed above to prevent pregnancy, local regulations apply and will be described in the ICF 6. Active systemic bacterial, viral (incl. COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration 7. History of recurrent invasive infections caused by encapsulated organisms, e.g N. meningitidis and S. pneumoniae 8. Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration 9. History of autoimmune disease other than MG (e.g., thyroiditis, rheumatoid arthritis, etc.) that would interfere with an accurate assessment of clinical symptoms 10. Documented lack of clinical response to PLEX 11. Participants with clinical evidence of other significant serious disease or patients who underwent a recent major surgery, which could confound the results of the trial or put the patient at undue risk 12. Participation in any other investigational drug trial or use of other investigational drugs at the time of enrollment, or within 5 elimination half-lives of enrollment, or within 30 days of enrollment whichever is longer; or longer if required by local regulations 13. History of current treatment for hepatic disease including but not limited to acute or chronic hepatitis, cirrhosis or hepatic failure, or aspartate aminotransferase (AST), alanine aminotransferase (ALT), gamma-glutamyl transferase (GGT) or alkaline phosphatase (ALP) PAT059532 FF of more than 3 x upper limit of normal (ULN) or serum bilirubin 2 x ULN (with the exception of those participants with a known confirmed diagnosis of Gilbert syndrome). 14. History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer), treated or untreated, within the past 5 years, regardless of whether there is evidence of local recurrence or metastases. 15. Participants with worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoroquinolones, beta-blockers, etc.) 16. History or current diagnosis, of ECG abnormalities indicating significant risk of safety for subjects participating in the study such as: ^ Concomitant clinically significant cardiac arrhythmias, eg sustained ventricular tachycardia, and clinically significant second or third degree AV block without a pacemaker ^ History of familial long QT syndrome or known family history of Torsades de Pointes ^ Resting heart rate (physical exam or 12 lead ECG) <60 bpm 17. History of T-lymphocyte or T-lymphocyte-receptor vaccination, transplantation (including solid organ, stem cell, and bone marrow transplantation) and/or antirejection therapy 18. Participants not responsive to eculizumab, ravulizumab, or other complement inhibitors 19. Known or suspected hereditary complement deficiency 20. Major concurrent comorbidities including but not limited to severe kidney disease (e.g., eGFR < 30 mL/min, dialysis), advanced cardiac disease (e.g., NYHA class IV), severe pulmonary disease (e.g., severe pulmonary hypertension) (WHO class IV), or hepatic disease (e.g., active hepatitis) 21. Ongoing drug or alcohol abuse that could interfere with patient's participation in the trial 22. Participants with a history of suicidal attempt or suicidal behavior within 6 months prior to screening and/or participants presenting suicidal ideation with intent documented by Columbia Suicidal Severity Rating Scale (CSSRS) by “Yes” response to Q4 or Q5 at screening Study Treatment(s) and concomitant therapy Study participants will be randomized to iptacopan 200 mg or placebo twice daily (b.i.d) in 1:1 ratio, while remaining on stable dose of pre-existing SOC treatment for gMG before baseline and throughout the trial as permitted and required per inclusion criteria. Treatment Duration PAT059532 FF The planned duration of randomized treatment is 6 months (180 days) for the Core part Double- blind Treatment Period. Study participants may be temporarily interrupted or discontinued from treatment earlier due to unacceptable toxicity, lack of efficacy (progression of underlying disease), intake of medication, bacterial infection and/or treatment is discontinued at the discretion of the investigator. If a participant's study treatment is discontinued for any reason, every effort must be made to continue with the study assessments up to Month 6 of the Double-blind Treatment Period. After completion of the 6-month Double-blind Treatment Period, participants will be offered to continue treatment in the Open-Label Extension and will receive iptacopan for 24 months. If a participant discontinues iptacopan treatment during the Open-label Extension, the investigator should follow the recommended procedures for discontinuation. Handling of other or additional treatment Subjects entering the study will be allowed to continue the SOC therapy outlined above. Moreover, the SOC therapy medications for gMG must be kept at the same dose throughout the study, including corticosteroids and IST drugs, unless rescue therapy becomes necessary. Rescue therapy may be administered due to deterioration of a subject’s clinical status, or in case of a risk of MG crisis as per the investigator judgment. In such cases, the subject may receive IVIG or PLEX treatment as rescue therapy. The SOC should be assigned as per local treatment policy at the discretion of the investigator and, as already mentioned, remain stable throughout the trial duration. The SOC medication use during the study will be recorded in participant's source document/medical chart and eCRF. No specific monitoring is required, except that stable SOC treatment need to be verified and documented on corresponding CRF pages. Dose modifications Iptacopan or matching placebo will be administered at 200 mg b.i.d. Investigational or other study treatment dose adjustments are not permitted. Dose escalations Iptacopan or matching placebo will be administered at 200 mg b.i.d. Investigational treatment dose escalations are not permitted. Concomitant therapies Immunosuppressants PAT059532 FF Patients who are receiving other immunosuppressive therapies (i.e., azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide), are required to be on these immunosuppressive therapies for at least 6 months and to have been on a stable dose prior to baseline and throughout the study (see inclusion/exclusion criteria). Cholinesterase Inhibitors For patients who enter the study receiving a cholinesterase inhibitor at screening, the dose and schedule of their cholinesterase inhibitor must be maintained stable throughout the entire Double- blind Treatment Period and Open-label Extension Period, unless there is compelling medical need. Increases in cholinesterase therapy that are required as a result of intercurrent illness or other medical cause of deterioration are permitted, but dosing must be returned to dosing levels at study entry as soon as feasible and the Sponsor should be notified of the change. 1. Cholinesterase inhibitor treatment must be withheld for at least 10 hours prior to administration of the QMG and MGC tests. 2. If a decrease in cholinesterase inhibitor is considered based on clinical evaluation, Sponsor approval must be obtained prior to the change in dose in order for the patient to remain on study. Corticosteroids Participants receiving oral corticosteroids are required to be on a stable dose for at least 4 weeks (i.e., 28 days or longer) prior to baseline. Equivalents Those skilled in the art will recognize, or be able to ascertain, using no more than routine experimentation, numerous equivalents to the specific embodiments described specifically herein. Such equivalents are intended to be encompassed in the scope of the following claims. Example 2: Iptacopan (LNP023) inhibits development of paralysis in the PTMG rat model. Example 2 presents the data obtained from a study conducted to evaluate the efficacy of LNP023 (iptacopan) in the PTMG model. The study involved a group of rats induced with severe PTMG using 1 mg/kg of mAb35. The results showed significant effects on disease onset, and maximal PAT059532 FF score and a trend for reduced body weight, indicating the potential of LNP023 as a therapeutic agent in PTMG. Methods Female Lewis rats aged 6 weeks were injected either with PBS (n=5, healthy controls) or a monoclonal antibody (rat IgG1) against acetylcholine receptor nicotinic alpha 1 subunit at 1 mg/kg (mAb35, n=25) via the intraperitoneal route. Prior to this, the mAb35-injected animals had been randomized to vehicle (disease controls, n=10) or LNP023 treatment (60 mg/kg, twice daily by oral gavage, n=15). Treatment was initiated one hour prior to injection of mAb35. The vehicle used was 0.5% MC / 0.5% Tween80. Animals were monitored twice daily for clinical symptoms and body weight changes. Clinical symptoms were scored as follows: 1: normal behavior, 2: mild weakness, completely flaccid tail, 3: significant weakness. At least two of the following symptoms: completely flaccid tail, hunched back, chin down. 4: severe weakness: no mobility in one or more limbs and/or breathing difficulty. Animal is euthanized on the same day. Score 5: moribund with little or no movement. Animal is euthanized immediately. 36h post injection of mAb35, the combined muscle action potential (cMAP) was measured in the left hind limb of anaesthetised animals. The sciatic CMAP responses were obtained by stimulating the sciatic nerve at supramaximal intensity (20 mA) with square-wave pulses of 0.1 ms duration at 10 Hz with a 10-30 s pause between trains. CMAP responses were acquired from each stimulation. CMAP of the 15^th stimulation was normalized against the CMAP from the 1^st stimulation. Statistical analysis was performed using GraphPad Prism version 10.1.2. using Kruskal Wallis test followed by Dunn’s multiple comparison test. For cMAP, Brown-Forsythe and Welch ANOVA test was used followed by Dunnett’s T3 multiple comparison test. * p<0.05. Results Injection of a rat antibody against acetylcholine receptor (AChR, mAb35, 1 mg/kg, i.p.) into female Lewis rats resulted in a rapid development of weakness in Lewis rats in 6 out of 10 vehicle-treated control rats (Figure 1A). First clinical symptoms were observed 24h post injection and the rats developed significant weakness (Score 3) by 48h post injection. The remaining rats did not develop any clinical symptoms except one rat that presented with mild PAT059532 FF weakness at one single time point (60h, lasting less than 12h). Clinical symptoms were associated with pronounced body weight loss in the affected animals (Figure 1B). By 72h post injection, 5 out of the 7 sick rats had recovered from the disease. Unexpectedly, 2 rats injected with PBS instead of mAb35 also developed mild transient weakness (score 2) for 1 and 2 time points (<12 h and <24h), respectively, but this was not associated with any weight loss. Treatment with LNP023 (60 mg/kg bid, started 1h prior to injection of mAb35) significantly reduced the incidence and severity of disease with a strong trend for reduced duration of clinical symptoms compared to the vehicle-treated rats (Figure 1 A-D).4/15 rats developed mild transient weakness, of which only 1/15 rats achieved significant weakness (score 3), which lasted less than 12h (summarized graphically in Figure 1A). After LNP023 treatment, disease was associated with slightly reduced body weight loss compared to the vehicle-treated group (Figure 1B). Disease duration was assessed by quantifying the number of times an animal had a clinical score of more than 1; thereby giving a measure of disease duration (Figure 1C). As shown in Figure 1D, treatment with LNP023 significantly reduced the severity of disease with animals having a lower maximum clinical score. Compound muscle action potential (cMAP) was measured in the left hind limb 36h post disease induction and was found to be significantly reduced in the vehicle treated animals compared to the healthy sham animals. Due to the high variability between animals, only a trend for improvement in cMAP was observed after treatment with LNP023 (Figure 1E). In addition, treatment with LNP023 significantly delayed the onset of disease compared to the vehicle-treated group (Figure 1F). Conclusions These data demonstrate that treatment with LNP023 (iptacopan) significantly reduces the incidence and severity of PTMG in Lewis rats.

Claims

PAT059532 FF CLAIMS 2. A method of treating generalized Myasthenia Gravis (gMG) in a subject in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. 3. The method according to claim 1, wherein the subject has anti-acetylcholine receptor antibody-positive (AChR+) gMG. 4. The method according to any one of claims 1 to 2, wherein the method further comprises achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 5. The method according to claim 3, wherein the reduction in a MG-ADL total score is by at least 2 points, in particular the reduction in a MG-ADL total score is by at least 2 points at 6 months compared to at baseline. 6. The method according to claim 4, wherein the reduction in a MG-ADL total score is by at least 3 points, in particular the reduction in a MG-ADL total score is by at least 3 points at 6 months compared to at baseline. 7. The method according to any one of claims 1 to 5, wherein the method further comprises achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 8. The method according to claim 6, wherein the reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. PAT059532 FF 9. The method according to claim 7, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. 10. The method according to any one of the preceding claims, wherein the method comprises one or more of the following: a. achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b. achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c. achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d. achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e. achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 11. A method of reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. 12. The method according to claim 10, wherein the method achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, e.g., at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. PAT059532 FF 13. The method according to claim 11, wherein the method achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, in particular the method achieves a reduction in a MG-ADL total score by at least 2 points at 6 months compared to at baseline. 14. The method according to claim 12, wherein the method achieves a reduction in a MG-ADL total score by at least 3 points compared to at baseline, in particular the method achieves a reduction in a MG-ADL total score by at least 3 points at 6 months compared to at baseline. 15. A method of reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject in need thereof, the method comprising administering to the subject iptacopan or a pharmaceutically acceptable salt or hydrate thereof. 16. The method according to claim 14, wherein the method achieves a reduction in a QMG total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 17. The method according to claim 15, wherein the method achieves a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the method achieves a reduction in a QMG total score by at least 4 points at 6 months compared to at baseline. 18. The method according to claim 16, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. 19. The method according to any one of claims 1 to 17, wherein the method comprises administering to the subject iptacopan hydrochloride. 20. The method according to claim 18, wherein the method comprises administering to the subject iptacopan hydrochloride monohydrate. PAT059532 FF 21. The method according to any one of claims 1 to 19, wherein the method comprises administering to the subject iptacopan at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, or from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). 22. The method according to any one of claims 1 to 20, wherein the method comprises administering to the subject iptacopan at a dose of about 50 mg, or about 75 mg, or about 100 mg, or about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). 23. The method according to any one of claims 1 to 21, wherein the dose is about 50 mg twice daily. 24. The method according to any one of claims 1 to 21, wherein the dose is about 100 mg twice daily. 25. The method according to any one of claims 1 to 21, wherein the dose is about 200 mg twice daily. 26. The method according to any one of claims 1 to 24, wherein iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. 27. The method according to any one of claims 1 to 25, wherein the method further comprises administering to the subject an immunosuppressant. 28. The method according to claim 26, wherein the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. 29. The method according to claim 26 or claim 27, wherein the subject has been administered the immunosuppressant for at least six months. PAT059532 FF 30. The method according to any one of claims 1 to 28, wherein the method further comprises administering to the subject a corticosteroid. 31. The method according to claim 29, wherein the subject has been administered a corticosteroid for at least four weeks. 32. The method according to any one of claims 1 to 30, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. 33. The method according to any one of claims 1 to 31, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis and Streptococcus pneumoniae. 34. The method according to any one of claims 1 to 32, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Haemophilus influenzae. 35. The method according to any one of claims 1 to 33, wherein the subject is not optimally controlled for greater than or equal to 6 months on one non-steroidal immunosuppressive therapies (NSIST). 36. The method according to any one of claims 1 to 33, wherein the subject is not optimally controlled for greater than or equal to 6 months on two or more non-steroidal immunosuppressive therapies (NSIST). 37. The method according to claim 34 or claim 35, wherein the NSIST is azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide. PAT059532 FF 38. The method according to any one of claims 1 to 36, wherein the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved for gMG. 39. The method according to any one of claims 1 to 37, wherein the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non-steroidal immunosuppressive therapies (NSISTs). 40. The method according to any one of claims 1 to 38, wherein the method further comprises administering to the subject azathioprine. 41. The method according to claim 39, wherein the method further comprises administering to the subject azathioprine for at least 6 months. 42. The method according to any one of claims 1 to 40, wherein the method further comprises administering to the subject a cholinesterase inhibitor. 43. The method according to claim 41, wherein the method further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. 44. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in a treatment of generalized Myasthenia Gravis (gMG) in a subject in need thereof. 45. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 43, wherein gMG is anti-acetylcholine receptor antibody-positive (AChR+) gMG. 46. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 44, wherein the use further comprises achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, PAT059532 FF at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 47. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 45, wherein the reduction in a MG-ADL total score is by at least 2 points compared to at baseline, in particular the reduction in a MG-ADL total score is by at least 2 points at 6 months compared to at baseline. 48. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 46, wherein the reduction in a MG-ADL total score is by at least 3 points compared to at baseline, in particular the reduction in a MG-ADL total score is by at least 3 points at 6 months compared to at baseline. 49. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 47, wherein the use further comprises achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 50. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 48, wherein the reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. 51. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 49, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. 52. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 50, wherein the use comprises one or more of the following: PAT059532 FF a. achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b. achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c. achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d. achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e. achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 53. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject in need thereof. 54. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 52, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points, e.g., at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 55. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 53, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, in particular the use achieves a reduction in a MG-ADL total score by at least 2 points at 6 months compared to at baseline. 56. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 54, wherein the use achieves a reduction in a MG-ADL total score by at least 3 points PAT059532 FF compared to at baseline, in particular the use achieves a reduction in a MG-ADL total score by at least 3 points at 6 months compared to at baseline. 57. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject in need thereof. 58. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 56, wherein the use achieves a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 59. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 57, wherein the use achieves a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the use achieves a reduction in a QMG total score by at least 4 points at 6 months compared to at baseline. 60. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 58, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. 61. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 59, wherein the use comprises administering to the subject iptacopan hydrochloride. 62. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 60, wherein the use comprises administering to the subject iptacopan hydrochloride monohydrate. PAT059532 FF 63. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 61, wherein the use comprises administering to the subject iptacopan at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, or from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). 64. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 62, wherein the use comprises administering to the subject iptacopan at a dose of about 50 mg, or about 75 mg, or about 100 mg, or about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). 65. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 63, wherein the dose is about 50 mg twice daily. 66. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 63, wherein the dose is about 100 mg twice daily. 67. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 63, wherein the dose is about 200 mg twice daily. 68. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 66, wherein iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. 69. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 67, wherein the use further comprises administering to the subject an immunosuppressant. 70. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 68, wherein the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. PAT059532 FF 71. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 68 or claim 69, wherein the subject has been administered the immunosuppressant for at least six months. 72. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 70, wherein the use further comprises administering to the subject a corticosteroid. 73. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 71, wherein the subject has been administered a corticosteroid for at least four weeks. 74. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 72, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. 75. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 73, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis and Streptococcus pneumoniae. 76. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 74, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Haemophilus influenzae. 77. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 75, wherein the subject is not optimally controlled for greater than or equal to 6 months on one non-steroidal immunosuppressive therapies (NSIST). PAT059532 FF 78. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 75, wherein the subject is not optimally controlled for greater than or equal to 6 months on two or more non-steroidal immunosuppressive therapies (NSIST). 79. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 76 or claim 77, wherein the NSIST is azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide. 80. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 78, wherein the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved from gMG. 81. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 79, wherein the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non- steroidal immunosuppressive therapies (NSISTs). 82. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 80, wherein the use further comprises administering to the subject azathioprine. 83. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 81, wherein the use further comprises administering to the subject azathioprine for at least 6 months. 84. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to any one of claims 43 to 82, wherein the method further comprises administering to the subject a cholinesterase inhibitor. PAT059532 FF 85. Iptacopan or a pharmaceutically acceptable salt or hydrate thereof for use according to claim 83, wherein the method further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. 86. A pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in treating generalized Myasthenia Gravis (gMG) in a subject in need thereof. 87. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 85, wherein gMG is anti-acetylcholine receptor antibody-positive (AChR+) gMG. 88. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 86, wherein the use further comprises achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 89. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 87, wherein the reduction in a MG-ADL total score is by at least 2 points compared to at baseline, in particular the reduction in a MG-ADL total score is by at least 2 points at 6 months compared to at baseline. 90. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 88, wherein the reduction in a MG-ADL total score is by at least 3 points compared to at baseline, in particular the reduction in a MG-ADL total score is by at least 3 points at 6 months compared to at baseline. PAT059532 FF 91. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 89, wherein the use further comprises achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 92. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 90, wherein the reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the reduction in a QMG total score is by at least 4 points at 6 months compared to at baseline. 93. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 91, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. 94. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of preceding claims 85 to 92, wherein the use comprises one or more of the following: a. achieving a reduction in a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score, e.g., by at least 2 points, at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; b. achieving a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least PAT059532 FF 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; c. achieving a reduction in a Myasthenia Gravis Composite (MGC) total score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; d. achieving a reduction in a MG-QOL15r survey score, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline; e. achieving an increase in a EQ-5D-5L survey score on EQ VAS scale, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 95. A pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Myasthenia Gravis Activity of Daily Living (MG-ADL) total score in a subject in need thereof. 96. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 94, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points, e.g., at least 3 points, at least 4 points, at least 5 points, or at least 6 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 97. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 95, wherein the use achieves a reduction in a MG-ADL total score by at least 2 points compared to at baseline, in particular the use achieves a reduction in a MG- ADL total score by at least 2 points at 6 months compared to at baseline. 98. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 96, wherein the use achieves a reduction in a MG-ADL total score by at least 3 points compared to at baseline, in particular the use achieves a reduction in a MG- ADL total score by at least 3 points at 6 months compared to at baseline. PAT059532 FF 99. A pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use in reducing a Quantitative Myasthenia Gravis (QMG) total score in a subject in need thereof. 100. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 98, wherein the use achieves a reduction in a Quantitative Myasthenia Gravis (QMG) total score, e.g., by at least 3 points, e.g., at least 4 points, at least 5 points, at least 6 points, at least 7 points, at least 8 points, at least 9 points, at least 10 points, e.g., at 1 month, at 3 months, at 6 months, compared to at baseline. 101. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 99, wherein the use achieves a reduction in a QMG total score is by at least 4 points compared to at baseline, in particular the use achieves a reduction in a QMG total score by at least 4 points at 6 months compared to at baseline. 102. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 100, wherein the reduction in a QMG total score is by at least 5 points compared to at baseline, in particular the reduction in a QMG total score is by at least 5 points at 6 months compared to at baseline. 103. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 101, wherein the use comprises administering to the subject iptacopan hydrochloride. 104. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for PAT059532 FF use according to claim 102, wherein the use comprises administering to the subject iptacopan hydrochloride monohydrate. 105. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 103, wherein the use comprises administering to the subject iptacopan at a dose of from about 50 mg to about 500 mg, e.g., from about 50 mg to about 250 mg, or from about 100 mg to about 200 mg, each administered twice daily (b.i.d.). 106. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 104, wherein the use comprises administering to the subject iptacopan at a dose of about 50 mg, or about 75 mg, or about 100 mg, or about 150 mg, or about 200 mg, each administered twice daily (b.i.d.). 107. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 105, wherein the dose is about 50 mg twice daily. 108. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 105, wherein the dose is about 100 mg twice daily. 109. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 105, wherein the dose is about 200 mg twice daily. 110. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for PAT059532 FF use according to any one of claims 85 to 108, wherein iptacopan or a pharmaceutically acceptable salt or hydrate thereof is administered orally. 111. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 109, wherein the use further comprises administering to the subject an immunosuppressant. 112. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 110, wherein the immunosuppressant is at least one of azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, and cyclophosphamide. 113. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 110 or claim 111, wherein the subject has been administered the immunosuppressant for at least six months. 114. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 112, wherein the use further comprises administering to the subject a corticosteroid. 115. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 113, wherein the subject has been administered a corticosteroid for at least four weeks. 116. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 114, wherein the subject has been vaccinated prior PAT059532 FF to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against at least one of Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae. 117. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 115, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Neisseria meningitidis and Streptococcus pneumoniae. 118. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 116, wherein the subject has been vaccinated prior to administration of iptacopan or a pharmaceutically acceptable salt or hydrate thereof against Haemophilus influenzae. 119. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 117, wherein the subject is not optimally controlled for greater than or equal to 6 months on one non-steroidal immunosuppressive therapies (NSIST). 120. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 117, wherein the subject is not optimally controlled for greater than or equal to 6 months on two or more non-steroidal immunosuppressive therapies (NSIST). 121. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for PAT059532 FF use according to claim 118 or claim 119, wherein the NSIST is azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, tacrolimus, or cyclophosphamide. 122. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 120, wherein the subject is not optimally controlled for greater than or equal to 6 months on a gMG treatment that is rituximab or a neonatal crystallizable fragment receptor (FcRN) antagonist approved for gMG. 123. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 121, wherein the subject is not optimally controlled for greater than or equal to 6 months on frequent (e.g., at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and non-steroidal immunosuppressive therapies (NSISTs). 124. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 122, wherein the use further comprises administering to the subject azathioprine. 125. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 123, wherein the use further comprises administering to the subject azathioprine for at least 6 months. 126. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to any one of claims 85 to 124, wherein the use further comprises administering to the subject a cholinesterase inhibitor. PAT059532 FF 127. The pharmaceutical composition comprising iptacopan or a pharmaceutically acceptable salt or hydrate thereof and at least one pharmaceutically acceptable carrier for use according to claim 125, wherein the use further comprises administering to the subject the cholinesterase inhibitor for at least two weeks. 128. Use of iptacopan or a pharmaceutically acceptable salt or hydrate thereof for the manufacture of a medicament for treating generalized Myasthenia Gravis (gMG) in a subject in need thereof.