CA3167476A1 - Methods of treating fabry disease - Google Patents
Methods of treating fabry disease Download PDFInfo
- Publication number
- CA3167476A1 CA3167476A1 CA3167476A CA3167476A CA3167476A1 CA 3167476 A1 CA3167476 A1 CA 3167476A1 CA 3167476 A CA3167476 A CA 3167476A CA 3167476 A CA3167476 A CA 3167476A CA 3167476 A1 CA3167476 A1 CA 3167476A1
- Authority
- CA
- Canada
- Prior art keywords
- patient
- migalastat
- assessing
- therapy
- ert
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
- A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
- A61K31/445—Non condensed piperidines, e.g. piperocaine
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
- A61B5/00—Measuring for diagnostic purposes; Identification of persons
- A61B5/42—Detecting, measuring or recording for evaluating the gastrointestinal, the endocrine or the exocrine systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61B—DIAGNOSIS; SURGERY; IDENTIFICATION
- A61B5/00—Measuring for diagnostic purposes; Identification of persons
- A61B5/48—Other medical applications
- A61B5/4848—Monitoring or testing the effects of treatment, e.g. of medication
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Urology & Nephrology (AREA)
- Heart & Thoracic Surgery (AREA)
- Epidemiology (AREA)
- Vascular Medicine (AREA)
- Cardiology (AREA)
- Medical Informatics (AREA)
- Surgery (AREA)
- Molecular Biology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Pathology (AREA)
- Biomedical Technology (AREA)
- Physiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Endocrinology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Obesity (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062972392P | 2020-02-10 | 2020-02-10 | |
| US62/972,392 | 2020-02-10 | ||
| PCT/US2021/017446 WO2021163183A1 (en) | 2020-02-10 | 2021-02-10 | Methods of treating fabry disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3167476A1 true CA3167476A1 (en) | 2021-08-19 |
Family
ID=77272302
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3167476A Pending CA3167476A1 (en) | 2020-02-10 | 2021-02-10 | Methods of treating fabry disease |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20210251972A1 (https=) |
| EP (2) | EP4732901A2 (https=) |
| JP (1) | JP2023513700A (https=) |
| KR (1) | KR20220150902A (https=) |
| AU (1) | AU2021218578A1 (https=) |
| CA (1) | CA3167476A1 (https=) |
| DK (1) | DK4103178T3 (https=) |
| FI (1) | FI4103178T3 (https=) |
| LT (1) | LT4103178T (https=) |
| PT (1) | PT4103178T (https=) |
| WO (1) | WO2021163183A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3224546C (en) | 2017-05-30 | 2026-02-24 | Amicus Therapeutics, Inc. | Use of migalastat in the treatment of fabry patients |
| KR20200128676A (ko) | 2018-02-06 | 2020-11-16 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 |
| US12594268B2 (en) * | 2018-08-20 | 2026-04-07 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
| KR20220044560A (ko) | 2019-08-07 | 2022-04-08 | 아미쿠스 세라퓨틱스, 인코포레이티드 | Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법 |
| US11623916B2 (en) | 2020-12-16 | 2023-04-11 | Amicus Therapeutics, Inc. | Highly purified batches of pharmaceutical grade migalastat and methods of producing the same |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6274597B1 (en) | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
| MX2009010557A (es) | 2007-03-30 | 2009-11-19 | Amicus Therapeutics Inc | Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas. |
| US9999618B2 (en) * | 2007-04-26 | 2018-06-19 | Amicus Therapeutics, Inc. | Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones |
| EP2946785B1 (en) | 2008-02-12 | 2018-10-24 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| TW202408513A (zh) * | 2016-07-19 | 2024-03-01 | 美商阿米庫斯醫療股份有限公司 | 治療ert初治的和已經歷ert的患者中之法布瑞氏症 |
-
2021
- 2021-02-10 KR KR1020227030895A patent/KR20220150902A/ko active Pending
- 2021-02-10 WO PCT/US2021/017446 patent/WO2021163183A1/en not_active Ceased
- 2021-02-10 LT LTEPPCT/US2021/017446T patent/LT4103178T/lt unknown
- 2021-02-10 JP JP2022548459A patent/JP2023513700A/ja active Pending
- 2021-02-10 DK DK21753606.9T patent/DK4103178T3/da active
- 2021-02-10 PT PT217536069T patent/PT4103178T/pt unknown
- 2021-02-10 CA CA3167476A patent/CA3167476A1/en active Pending
- 2021-02-10 AU AU2021218578A patent/AU2021218578A1/en active Pending
- 2021-02-10 US US17/172,846 patent/US20210251972A1/en active Pending
- 2021-02-10 EP EP25226333.0A patent/EP4732901A2/en active Pending
- 2021-02-10 FI FIEP21753606.9T patent/FI4103178T3/fi active
- 2021-02-10 EP EP21753606.9A patent/EP4103178B1/en active Active
Also Published As
| Publication number | Publication date |
|---|---|
| LT4103178T (lt) | 2026-04-10 |
| EP4732901A2 (en) | 2026-04-29 |
| FI4103178T3 (fi) | 2026-03-23 |
| EP4103178A1 (en) | 2022-12-21 |
| DK4103178T3 (da) | 2026-03-30 |
| EP4103178B1 (en) | 2025-12-24 |
| AU2021218578A1 (en) | 2022-08-25 |
| PT4103178T (pt) | 2026-03-30 |
| US20210251972A1 (en) | 2021-08-19 |
| EP4103178A4 (en) | 2024-03-20 |
| KR20220150902A (ko) | 2022-11-11 |
| WO2021163183A1 (en) | 2021-08-19 |
| JP2023513700A (ja) | 2023-04-03 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| EP3851105B1 (en) | Migalastat for use in treating fabry disease in patients having a mutation in the gla gene | |
| EP4103178B1 (en) | Methods of treating fabry disease | |
| US12599594B2 (en) | Treatment of Fabry disease in ERT-naïve and ERT-experienced patients | |
| US11426396B2 (en) | Methods of treating Fabry patients having renal impairment | |
| EP4176877B1 (en) | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease | |
| US20220313670A1 (en) | Methods Of Treating Fabry Disease In Patients Having Renal Impairment | |
| US20240293387A1 (en) | Methods of Treating Fabry Disease in Pediatric Patients | |
| US20220087993A1 (en) | Use of migalostat in reducing the risk of cerebrovascular event in patients with fabry disease | |
| US20260076951A1 (en) | Methods of treating fabry disease in patients having renal impairment | |
| EP4385509A1 (en) | Migalastat formulations having improved pharmacokinetics | |
| HK40128496A (en) | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease | |
| HK40124728A (en) | Methods of reducing cerebrovascular events in patients with fabry disease | |
| HK40106025A (en) | Treatment of patients with classic fabry disease |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| EEER | Examination request |
Effective date: 20220929 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION Effective date: 20240719 |
|
| B12 | Application deemed to be withdrawn, abandoned or lapsed |
Free format text: ST27 STATUS EVENT CODE: N-2-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION Effective date: 20240916 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: N-6-6-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT Effective date: 20241011 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 4TH ANNIV.) - STANDARD Year of fee payment: 4 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20241220 |
|
| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: N-6-6-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20250212 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: N-6-6-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20250212 |
|
| D15 | Examination report completed |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT Effective date: 20250722 |
|
| P11 | Amendment of application requested |
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| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD Year of fee payment: 5 |
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| U00 | Fee paid |
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| U11 | Full renewal or maintenance fee paid |
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| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT Effective date: 20260106 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION AMENDED Effective date: 20260106 |
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| U11 | Full renewal or maintenance fee paid |
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| W00 | Other event occurred |
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|
| D15 | Examination report completed |
Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT Effective date: 20260420 |