JP2023509520A - 発作性夜間ヘモグロビン尿症の治療 - Google Patents

発作性夜間ヘモグロビン尿症の治療 Download PDF

Info

Publication number
JP2023509520A
JP2023509520A JP2022541894A JP2022541894A JP2023509520A JP 2023509520 A JP2023509520 A JP 2023509520A JP 2022541894 A JP2022541894 A JP 2022541894A JP 2022541894 A JP2022541894 A JP 2022541894A JP 2023509520 A JP2023509520 A JP 2023509520A
Authority
JP
Japan
Prior art keywords
subject
pegcetacoplan
level
weeks
target
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
Application number
JP2022541894A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023509520A5 (cg-RX-API-DMAC10.html
Inventor
デシャテレッツ，パスカル
フランソワ，セドリック
グロッシ，フェデリコ
モリス，シャロン
エフ タン，エリザベス
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Apellis Pharmaceuticals Inc
Original Assignee
Apellis Pharmaceuticals Inc
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Apellis Pharmaceuticals Inc filed Critical Apellis Pharmaceuticals Inc
Publication of JP2023509520A publication Critical patent/JP2023509520A/ja
Publication of JP2023509520A5 publication Critical patent/JP2023509520A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/04—Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/12—Cyclic peptides, e.g. bacitracins; Polymyxins; Gramicidins S, C; Tyrocidins A, B or C
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/395—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/3955—Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against proteinaceous materials, e.g. enzymes, hormones, lymphokines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/56—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/26—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving oxidoreductase
    • C12Q1/32—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving oxidoreductase involving dehydrogenase
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/72—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving blood pigments, e.g. haemoglobin, bilirubin or other porphyrins; involving occult blood
    • G01N33/721—Haemoglobin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/545—Medicinal preparations containing antigens or antibodies characterised by the dose, timing or administration schedule
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K7/00—Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/50—Cyclic peptides containing at least one abnormal peptide link
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/22—Haematology
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/52—Predicting or monitoring the response to treatment, e.g. for selection of therapy based on assay results in personalised medicine; Prognosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Urology & Nephrology (AREA)
  • Physics & Mathematics (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Analytical Chemistry (AREA)
  • Mycology (AREA)
  • Endocrinology (AREA)
  • Food Science & Technology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Pathology (AREA)
  • General Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
JP2022541894A 2020-01-07 2021-01-07 発作性夜間ヘモグロビン尿症の治療 Pending JP2023509520A (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202062958265P 2020-01-07 2020-01-07
US62/958,265 2020-01-07
US202062961032P 2020-01-14 2020-01-14
US62/961,032 2020-01-14
US202063038607P 2020-06-12 2020-06-12
US63/038,607 2020-06-12
US202063080648P 2020-09-18 2020-09-18
US63/080,648 2020-09-18
US202063124006P 2020-12-10 2020-12-10
US63/124,006 2020-12-10
PCT/US2021/012561 WO2021142171A1 (en) 2020-01-07 2021-01-07 Treatment of paroxysmal nocturnal hemoglobinuria

Publications (2)

Publication Number Publication Date
JP2023509520A true JP2023509520A (ja) 2023-03-08
JP2023509520A5 JP2023509520A5 (cg-RX-API-DMAC10.html) 2024-01-16

Family

ID=76788458

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022541894A Pending JP2023509520A (ja) 2020-01-07 2021-01-07 発作性夜間ヘモグロビン尿症の治療

Country Status (7)

Country Link
US (1) US20230076527A1 (cg-RX-API-DMAC10.html)
EP (1) EP4087541A4 (cg-RX-API-DMAC10.html)
JP (1) JP2023509520A (cg-RX-API-DMAC10.html)
CN (1) CN115243673A (cg-RX-API-DMAC10.html)
AU (1) AU2021206693A1 (cg-RX-API-DMAC10.html)
CA (1) CA3167017A1 (cg-RX-API-DMAC10.html)
WO (1) WO2021142171A1 (cg-RX-API-DMAC10.html)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4398926A4 (en) * 2021-09-09 2025-07-02 Apellis Pharmaceuticals Inc TREATMENT OF GEOGRAPHIC ATROPHY

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112019020955A2 (pt) * 2017-04-07 2020-05-05 Apellis Pharmaceuticals, Inc. regimes de dosagem e composições e métodos relacionados
JP2021507884A (ja) * 2017-12-15 2021-02-25 アペリス・ファーマシューティカルズ・インコーポレイテッドＡｐｅｌｌｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 投与レジメンならびに関連組成物および方法

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
"Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinu", [ONLINE], JPN6024053582, December 2019 (2019-12-01), ISSN: 0005722412 *
BERENTSEN, S. ET AL.: "Novel insights into the treatment of complement-mediated hemolytic anemias", THERAPEUTIC ADVANCES IN HEMATOLOGY, vol. 10, JPN6024053581, 2019, pages 1 - 20, ISSN: 0005722411 *
RISITANO, A. M. ET AL.: "Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhi", FRONTIERS IN IMMUNOLOGY, vol. 10, JPN6024053579, 2019, pages 1157, ISSN: 0005722409 *
WONG, R. S. M. ET AL.: "Inhibition of C3 with APL-2 Results in Normalisation of Markers of Intravascular and Extravascular H", BLOOD, vol. Vol.132, Suppl.1, JPN6024053580, 2018, pages 2314, ISSN: 0005722410 *

Also Published As

Publication number Publication date
WO2021142171A1 (en) 2021-07-15
CA3167017A1 (en) 2021-07-15
EP4087541A1 (en) 2022-11-16
US20230076527A1 (en) 2023-03-09
AU2021206693A1 (en) 2022-08-25
EP4087541A4 (en) 2024-03-27
CN115243673A (zh) 2022-10-25

Similar Documents

Publication Publication Date Title
Vargason et al. The evolution of commercial drug delivery technologies
ES3056525T3 (en) High concentration vegf receptor fusion protein containing formulations
CN102946861B (zh) 浓缩蛋白制剂及其用途
US20150150982A1 (en) Pharmaceutical formulation for a therapeutic antibody
BR112020001703B1 (pt) Solução aquosa estável compreendendo um anticorpo anti-c5, método para produção desta solução, uso da mesma para tratar uma condição associada ao complemento e kit terapêutico compreendendo a mesma
EP3988083B1 (en) Long-acting growth hormone dosage forms
CN108420788A (zh) 含有抗pcsk9抗体的稳定配制剂
KR20200123091A (ko) 투여 요법 및 관련 조성물 및 방법
CN108026175A (zh) 用于治疗癫痫的方法
ES2926627T3 (es) Composición farmacéutica para tratar la deficiencia de hormona del crecimiento pediátrica que contiene proteína de fusión de hGH
US20190240345A1 (en) Novel stable formulation
US20230076527A1 (en) Treatment of paroxysmal nocturnal hemoglobinuria
JP2024537284A (ja) 抗ｎｐｒ１抗体を投与することにより血行動態の変化を引き起こす方法
JP6516855B2 (ja) Ｃ１エステラーゼ阻害剤の医薬製剤
US20260069653A1 (en) Complement inhibitor dosing regimens
HK40082135A (en) Treatment of paroxysmal nocturnal hemoglobinuria
CN118056572A (zh) 含有抗il-17抗体的稳定制剂
CN112423790B (zh) 包含结合pcsk9的分子的组合物和使用方法
ES2779126T3 (es) Uso de un inhibidor de PCSK9 para tratar hiperlipidemia
US20250000936A1 (en) Treatment of geographic atrophy
HK40106296A (en) High concentration vegf receptor fusion protein containing formulations
US20230381269A1 (en) Methods of treating eye disorders
JP2019525931A (ja) ヒト成長ホルモンアナログによる成人成長ホルモン欠乏症の処置
US20210023236A1 (en) Multi-targeted, tunable, sustained delivery of payloads to charged avascular tissues
HK40106296B (en) High concentration vegf receptor fusion protein containing formulations

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20221012

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20240105

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20240105

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20241119

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20250107

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250404

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250606

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20250704

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20251104

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260303

A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20260728