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
pregnancy
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JP2021512906A5 (enExample
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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)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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 (enExample) 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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US (2) US11357784B2 (enExample)
EP (2) EP4717319A3 (enExample)
JP (2) JP7555818B2 (enExample)
KR (1) KR20200128676A (enExample)
CN (1) CN111971044A (enExample)
AU (2) AU2019217868A1 (enExample)
CA (1) CA3090496C (enExample)
ES (1) ES3065184T3 (enExample)
WO (1) WO2019157047A1 (enExample)

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)

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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

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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
JP5270161B2 (ja) * 2004-09-24 2013-08-21 ベス イスラエル デアコネス メディカル センター 妊娠合併症を診断および処置する方法
PL2027137T3 (pl) 2005-06-08 2015-08-31 Amicus Therapeutics Inc Oczyszczanie imino- i aminocukrów
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
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
AU2008245578A1 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2009102895A2 (en) 2008-02-12 2009-08-20 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
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
EP2502068B1 (en) 2009-11-17 2016-02-10 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
WO2012125402A2 (en) 2011-03-11 2012-09-20 Amicus Therapeutics, Inc. Dosing regimens for the treatment of fabry disease
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
EP2874648A4 (en) 2012-07-17 2015-12-30 Amicus Therapeutics Inc ALPHA GALACTOSIDASE A AND 1-DEOXYGALACTONO JIRIMYCIN CO FORMULATION
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
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
AU2018277756A1 (en) 2017-05-30 2020-01-23 Amicus Therapeutics, Inc. Methods of treating fabry patients having renal impairment
WO2019046244A1 (en) 2017-08-28 2019-03-07 Amicus Therapeutics, Inc. METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE
AU2019217603A1 (en) 2018-02-06 2020-09-24 Amicus Therapeutics, Inc. Treatment of patients with classic Fabry disease
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
US20220313670A1 (en) 2019-06-11 2022-10-06 Amicus Therapeutics, Inc. Methods Of Treating Fabry Disease In Patients Having Renal Impairment
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
HRP20260399T1 (hr) 2020-02-10 2026-05-08 Amicus Therapeutics, Inc. Postupci liječenja fabryjeve bolesti

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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

Non-Patent Citations (1)

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CEN Case Rep，2017年，Vol.6，p.210-214

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Publication number Publication date
US11357784B2 (en) 2022-06-14
JP2024161361A (ja) 2024-11-19
KR20200128676A (ko) 2020-11-16
CA3090496C (en) 2024-03-26
EP4717319A3 (en) 2026-06-10
WO2019157047A1 (en) 2019-08-15
AU2019217868A1 (en) 2020-10-01
CN111971044A (zh) 2020-11-20
EP3749307A1 (en) 2020-12-16
US20210038624A1 (en) 2021-02-11
CA3090496A1 (en) 2019-08-15
EP4717319A2 (en) 2026-04-01
AU2024278298A1 (en) 2025-01-09
EP3749307B1 (en) 2025-12-10
ES3065184T3 (en) 2026-05-04
US20220387462A1 (en) 2022-12-08
JP2021512906A (ja) 2021-05-20

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