JP2021512896A - 古典型ファブリー病患者の治療 - Google Patents

古典型ファブリー病患者の治療 Download PDF

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Publication number
JP2021512896A
JP2021512896A JP2020542569A JP2020542569A JP2021512896A JP 2021512896 A JP2021512896 A JP 2021512896A JP 2020542569 A JP2020542569 A JP 2020542569A JP 2020542569 A JP2020542569 A JP 2020542569A JP 2021512896 A JP2021512896 A JP 2021512896A
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Prior art keywords
migalastat
salt
patient
effective amount
administration
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Japanese (ja)
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JPWO2019157056A5 (https=
JP2021512896A5 (https=
Inventor
ジェイ バース，
ジェイ バース，
エルフリダ ベンジャミン，
エルフリダ ベンジャミン，
Original Assignee
アミカス セラピューティックス インコーポレイテッド
アミカス セラピューティックス インコーポレイテッド
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Application filed by アミカス セラピューティックス インコーポレイテッド, アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2021512896A publication Critical patent/JP2021512896A/ja
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Priority to JP2023192942A priority Critical patent/JP2024026080A/ja
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    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/20—Pills, tablets, discs, rods
    • 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
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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

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  • Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2020542569A 2018-02-06 2019-02-06 古典型ファブリー病患者の治療 Withdrawn JP2021512896A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2023192942A JP2024026080A (ja) 2018-02-06 2023-11-13 古典型ファブリー病患者の治療

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862626992P 2018-02-06 2018-02-06
US62/626,992 2018-02-06
PCT/US2019/016853 WO2019157056A1 (en) 2018-02-06 2019-02-06 Treatment of patients with classic fabry disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2023192942A Division JP2024026080A (ja) 2018-02-06 2023-11-13 古典型ファブリー病患者の治療

Publications (3)

Publication Number Publication Date
JP2021512896A true JP2021512896A (ja) 2021-05-20
JPWO2019157056A5 JPWO2019157056A5 (https=) 2023-06-06
JP2021512896A5 JP2021512896A5 (https=) 2023-06-06

Family

ID=65494622

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2020542569A Withdrawn JP2021512896A (ja) 2018-02-06 2019-02-06 古典型ファブリー病患者の治療
JP2023192942A Pending JP2024026080A (ja) 2018-02-06 2023-11-13 古典型ファブリー病患者の治療

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023192942A Pending JP2024026080A (ja) 2018-02-06 2023-11-13 古典型ファブリー病患者の治療

Country Status (20)

Country Link
US (1) US20210038579A1 (https=)
EP (2) EP4316589A3 (https=)
JP (2) JP2021512896A (https=)
KR (1) KR20200128675A (https=)
CN (1) CN112203657A (https=)
AR (1) AR114392A1 (https=)
AU (2) AU2019217603A1 (https=)
DK (1) DK3749308T3 (https=)
ES (1) ES2969263T3 (https=)
FI (1) FI3749308T3 (https=)
HR (1) HRP20240025T1 (https=)
HU (1) HUE064761T2 (https=)
LT (1) LT3749308T (https=)
PL (1) PL3749308T3 (https=)
PT (1) PT3749308T (https=)
RS (1) RS65103B1 (https=)
SI (1) SI3749308T1 (https=)
SM (1) SMT202400027T1 (https=)
TW (2) TWI869336B (https=)
WO (1) WO2019157056A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2573498T3 (es) 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
CA3224537C (en) 2017-05-30 2025-09-09 Amicus Therapeutics, Inc. USE OF MIGALASTAT IN THE TREATMENT OF PATIENTS WITH FABRY DISEASE AND RENAL FAILURE
CA3090496C (en) 2018-02-06 2024-03-26 Amicus Therapeutics, Inc. Use of migalastat for treating fabry disease in pregnant patients
EP4282472A3 (en) 2018-08-20 2024-02-21 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene
EP3851105B1 (en) 2019-08-07 2026-03-04 Amicus Therapeutics, Inc. Migalastat for use in treating fabry disease in patients having a mutation in the gla gene
US11623916B2 (en) 2020-12-16 2023-04-11 Amicus Therapeutics, Inc. Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018017721A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients

Family Cites Families (6)

* Cited by examiner, † Cited by third party
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
ES2573498T3 (es) * 2006-05-16 2016-06-08 Amicus Therapeutics, Inc. Opciones de tratamiento para la enfermedad de Fabry
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
KR102004114B1 (ko) * 2016-03-22 2019-07-26 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
WO2019017938A1 (en) * 2017-07-19 2019-01-24 Amicus Therapeutics, Inc. TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018017721A1 (en) * 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients

Also Published As

Publication number Publication date
SMT202400027T1 (it) 2024-03-13
HUE064761T2 (hu) 2024-04-28
CN112203657A (zh) 2021-01-08
ES2969263T3 (es) 2024-05-17
WO2019157056A8 (en) 2020-12-03
DK3749308T3 (da) 2024-01-22
RS65103B1 (sr) 2024-02-29
EP4316589A3 (en) 2024-04-24
SI3749308T1 (sl) 2024-03-29
WO2019157056A1 (en) 2019-08-15
AU2019217603A1 (en) 2020-09-24
EP3749308A1 (en) 2020-12-16
JP2024026080A (ja) 2024-02-28
CA3090499A1 (en) 2019-08-15
KR20200128675A (ko) 2020-11-16
HRP20240025T1 (hr) 2024-03-29
FI3749308T3 (fi) 2024-01-18
US20210038579A1 (en) 2021-02-11
TWI869336B (zh) 2025-01-11
EP3749308B1 (en) 2023-10-25
AR114392A1 (es) 2020-09-02
LT3749308T (lt) 2024-02-12
EP4316589A2 (en) 2024-02-07
PT3749308T (pt) 2024-01-25
TW201944998A (zh) 2019-12-01
AU2025200074A1 (en) 2025-01-30
PL3749308T3 (pl) 2024-03-25
TW202537622A (zh) 2025-10-01

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