JP7555818B2 - 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用 - Google Patents

妊娠患者におけるファブリー病の治療のためのミガラスタットの使用 Download PDF

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JP7555818B2
JP7555818B2 JP2020542621A JP2020542621A JP7555818B2 JP 7555818 B2 JP7555818 B2 JP 7555818B2 JP 2020542621 A JP2020542621 A JP 2020542621A JP 2020542621 A JP2020542621 A JP 2020542621A JP 7555818 B2 JP7555818 B2 JP 7555818B2
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migalastat
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JP2021512906A (ja
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ジェイ バース，
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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
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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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008—Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • 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
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Urology & Nephrology (AREA)
  • Molecular Biology (AREA)
  • Dermatology (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2020542621A 2018-02-06 2019-02-06 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用 Active JP7555818B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024112288A JP2024161361A (ja) 2018-02-06 2024-07-12 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862626955P 2018-02-06 2018-02-06
US62/626,955 2018-02-06
PCT/US2019/016841 WO2019157047A1 (en) 2018-02-06 2019-02-06 Use of migalastat for treating fabry disease in pregnant patients

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JP2024112288A Division JP2024161361A (ja) 2018-02-06 2024-07-12 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用

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JP2021512906A JP2021512906A (ja) 2021-05-20
JP2021512906A5 JP2021512906A5 (https=) 2022-02-15
JPWO2019157047A5 JPWO2019157047A5 (https=) 2022-02-15
JP7555818B2 true JP7555818B2 (ja) 2024-09-25

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JP2020542621A Active JP7555818B2 (ja) 2018-02-06 2019-02-06 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用
JP2024112288A Pending JP2024161361A (ja) 2018-02-06 2024-07-12 妊娠患者におけるファブリー病の治療のためのミガラスタットの使用

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Country Link
US (2) US11357784B2 (https=)
EP (2) EP4717319A2 (https=)
JP (2) JP7555818B2 (https=)
KR (1) KR20200128676A (https=)
CN (1) CN111971044A (https=)
AU (2) AU2019217868A1 (https=)
CA (1) CA3090496C (https=)
WO (1) WO2019157047A1 (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)

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WO2018017721A1 (en) 2016-07-19 2018-01-25 Amicus Therapeutics, Inc. Treatment of fabry disease in ert-naïve and ert-experienced patients

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US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
SG155257A1 (en) * 2004-09-24 2009-09-30 Beth Israel Hospital Methods of diagnosing and treating complications of pregnancy
PL2027137T3 (pl) 2005-06-08 2015-08-31 Amicus Therapeutics Inc Oczyszczanie imino- i aminocukrów
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
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2150254A4 (en) 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
US8321148B2 (en) 2008-10-24 2012-11-27 Amicus Therapeutics, Inc. Multiple compartment dosing model
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
JP5739898B2 (ja) 2009-11-17 2015-06-24 ベイラー リサーチ インスティテュートＢａｙｌｏｒ Ｒｅｓｅａｒｃｈ Ｉｎｓｔｉｔｕｔｅ 心疾患マーカーとしての尿中トリアオシルセラミド（ｇｂ３）
CN103974619B (zh) 2011-03-11 2017-02-15 阿米库斯治疗学公司 治疗法布里病的给药方案
US10155027B2 (en) 2012-07-17 2018-12-18 Amicus Therapeutics, Inc. Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease
HK1209625A1 (en) 2012-07-17 2016-04-08 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
KR102004114B1 (ko) 2016-03-22 2019-07-26 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
TW201829770A (zh) 2017-01-10 2018-08-16 美商阿米庫斯醫療股份有限公司 用於治療fabry氏病之重組α-半乳糖苷酶A
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
IL272893B2 (en) 2017-08-28 2024-07-01 Amicus Therapeutics Inc Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
SMT202400027T1 (it) 2018-02-06 2024-03-13 Amicus Therapeutics Inc Trattamento di pazienti con malattia di fabry classica con migalastat
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
AR120055A1 (es) 2019-06-11 2022-02-02 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal
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
FI4103178T3 (fi) 2020-02-10 2026-03-23 Amicus Therapeutics Inc Menetelmiä fabryn taudin hoitamiseksi

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Also Published As

Publication number Publication date
EP3749307B1 (en) 2025-12-10
JP2021512906A (ja) 2021-05-20
WO2019157047A1 (en) 2019-08-15
CA3090496A1 (en) 2019-08-15
AU2019217868A1 (en) 2020-10-01
US20210038624A1 (en) 2021-02-11
US20220387462A1 (en) 2022-12-08
EP3749307A1 (en) 2020-12-16
EP4717319A2 (en) 2026-04-01
US11357784B2 (en) 2022-06-14
CA3090496C (en) 2024-03-26
KR20200128676A (ko) 2020-11-16
JP2024161361A (ja) 2024-11-19
AU2024278298A1 (en) 2025-01-09
CN111971044A (zh) 2020-11-20

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