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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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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migalastat
salt
patient
effective amount
administration
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JP2021512896A5 (enExample
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，
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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
Publication of JP2021512896A5 publication Critical patent/JP2021512896A5/ja
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)

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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 (2)

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JP2021512896A true JP2021512896A (ja) 2021-05-20
JP2021512896A5 JP2021512896A5 (enExample) 2023-06-06

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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

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

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024525760A (ja) * 2021-07-12 2024-07-12 アミカス セラピューティックス インコーポレイテッド 小児患者のファブリー病を治療する方法

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX351004B (es) 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
EP4717319A3 (en) 2018-02-06 2026-06-10 Amicus Therapeutics, Inc. Methods of treating fabry disease in pregnant patients
LT3840753T (lt) 2018-08-20 2024-11-11 Amicus Therapeutics, Inc. Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją
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

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
MX351004B (es) * 2006-05-16 2017-09-26 Nat Institutes Of Health Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry.
EP2142197A4 (en) 2007-03-30 2010-11-10 Amicus Therapeutics Inc METHOD FOR THE TREATMENT OF MORBUS FABRY WITH PHARMACOLOGICAL CHAPTERONES
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
EP3432882B1 (en) * 2016-03-22 2021-09-01 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
EA202090242A1 (ru) * 2017-07-19 2020-05-26 Амикус Терапьютикс, Инк. Лечение болезни фабри у фзт-наивных и фзт-леченных пациентов

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

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2024525760A (ja) * 2021-07-12 2024-07-12 アミカス セラピューティックス インコーポレイテッド 小児患者のファブリー病を治療する方法

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

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