KR20200128676A - 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 - Google Patents

임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 Download PDF

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Publication number
KR20200128676A
KR20200128676A KR1020207025691A KR20207025691A KR20200128676A KR 20200128676 A KR20200128676 A KR 20200128676A KR 1020207025691 A KR1020207025691 A KR 1020207025691A KR 20207025691 A KR20207025691 A KR 20207025691A KR 20200128676 A KR20200128676 A KR 20200128676A
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KR
South Korea
Prior art keywords
patient
migalastat
pregnancy
formulation
administered
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KR1020207025691A
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English (en)
Korean (ko)
Inventor
제이 바스
Original Assignee
아미쿠스 세라퓨틱스, 인코포레이티드
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Application filed by 아미쿠스 세라퓨틱스, 인코포레이티드 filed Critical 아미쿠스 세라퓨틱스, 인코포레이티드
Publication of KR20200128676A publication Critical patent/KR20200128676A/ko
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7008Compounds having an amino group directly attached to a carbon atom of the saccharide radical, e.g. D-galactosamine, ranimustine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/48Preparations in capsules, e.g. of gelatin, of chocolate
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs 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)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Urology & Nephrology (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)
KR1020207025691A 2018-02-06 2019-02-06 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도 Pending KR20200128676A (ko)

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

Publications (1)

Publication Number Publication Date
KR20200128676A true KR20200128676A (ko) 2020-11-16

Family

ID=65494618

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020207025691A Pending KR20200128676A (ko) 2018-02-06 2019-02-06 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도

Country Status (8)

Country Link
US (2) US11357784B2 (https=)
EP (2) EP3749307B1 (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
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
US12594268B2 (en) * 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
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

Family Cites Families (25)

* 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
EP2347765B1 (en) * 2004-09-24 2014-01-22 Beth Israel Deaconess Medical Center Methods of diagnosing and treating complications of pregnancy
SI2027137T1 (sl) 2005-06-08 2015-06-30 Amicus Therapeutics, Inc. Čiščenje imino in amino sladkorja
PL2533050T6 (pl) 2006-05-16 2016-05-31 Amicus Therapeutics Inc Możliwości leczenia choroby Fabry'ego
MX2009010557A (es) 2007-03-30 2009-11-19 Amicus Therapeutics Inc Metodo para el tratamiento de la enfermedad de fabry que utiliza chaperonas farmacologicas.
MX2009011473A (es) 2007-04-26 2010-01-18 Amicus Therapeutics Inc Regimenes de dosificacion para el tratamiento de enfermedades por almacenamiento lisosomal que utilizan chaperonas farmacologicas.
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
EP2946785B1 (en) 2008-02-12 2018-10-24 Amicus Therapeutics, Inc. Method to predict response to pharmacological chaperone treatment of diseases
WO2010048532A1 (en) 2008-10-24 2010-04-29 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
WO2011063048A2 (en) 2009-11-17 2011-05-26 Baylor Research Institute Urinary triaosylceramide (gb3) as a marker of cardiac disease
SG10201604757RA (en) * 2011-03-11 2016-08-30 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
WO2014014938A1 (en) 2012-07-17 2014-01-23 Amicus Therapeutics, Inc. Alpha-galactosidase a and 1-deoxygalactonojirimycin co-formulation
KR20190087673A (ko) 2016-03-22 2019-07-24 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 g9331a 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
TW202408513A (zh) * 2016-07-19 2024-03-01 美商阿米庫斯醫療股份有限公司 治療ert初治的和已經歷ert的患者中之法布瑞氏症
US11357834B2 (en) 2017-01-10 2022-06-14 Amicus Therapeutics, Inc. Recombinant α-galactosidase A for treatment of Fabry disease
CA3224546C (en) 2017-05-30 2026-02-24 Amicus Therapeutics, Inc. Use of migalastat in the treatment of fabry patients
US20200222377A1 (en) 2017-08-28 2020-07-16 Amicus Therapeutics, Inc. Methods Of Enhancing And/Or Stabilizing Cardiac Function In Patients With Fabry Disease
KR20200128676A (ko) 2018-02-06 2020-11-16 아미쿠스 세라퓨틱스, 인코포레이티드 임신 환자의 파브리병 치료를 위한 미갈라스타트의 용도
PT3749308T (pt) 2018-02-06 2024-01-25 Amicus Therapeutics Inc Tratamento de pacientes com doença de fabry clássica com migalastato
US12594268B2 (en) 2018-08-20 2026-04-07 Amicus Therapeutics, Inc. Methods of treating Fabry disease in patients having a mutation in the GLA gene
MX2021015352A (es) 2019-06-11 2022-04-06 Amicus Therapeutics Inc Métodos para tratar la enfermedad de fabry en pacientes que tienen insuficiencia renal.
KR20220044560A (ko) 2019-08-07 2022-04-08 아미쿠스 세라퓨틱스, 인코포레이티드 Gla 유전자에 돌연변이를 갖는 환자에서 파브리 질병을 치료하는 방법
KR20220150902A (ko) 2020-02-10 2022-11-11 아미쿠스 세라퓨틱스, 인코포레이티드 파브리 질병을 치료하는 방법

Also Published As

Publication number Publication date
US11357784B2 (en) 2022-06-14
WO2019157047A1 (en) 2019-08-15
JP7555818B2 (ja) 2024-09-25
EP4717319A2 (en) 2026-04-01
CN111971044A (zh) 2020-11-20
CA3090496C (en) 2024-03-26
AU2019217868A1 (en) 2020-10-01
JP2021512906A (ja) 2021-05-20
EP3749307A1 (en) 2020-12-16
JP2024161361A (ja) 2024-11-19
CA3090496A1 (en) 2019-08-15
US20210038624A1 (en) 2021-02-11
AU2024278298A1 (en) 2025-01-09
US20220387462A1 (en) 2022-12-08
EP3749307B1 (en) 2025-12-10

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