CN111971044A - 米加司他用于治疗妊娠患者的法布里病的用途 - Google Patents

米加司他用于治疗妊娠患者的法布里病的用途 Download PDF

Info

Publication number
CN111971044A
CN111971044A CN201980024425.7A CN201980024425A CN111971044A CN 111971044 A CN111971044 A CN 111971044A CN 201980024425 A CN201980024425 A CN 201980024425A CN 111971044 A CN111971044 A CN 111971044A
Authority
CN
China
Prior art keywords
patient
migalastat
pregnancy
formulation
treatment
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CN201980024425.7A
Other languages
English (en)
Chinese (zh)
Inventor
杰伊·巴斯
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Amicus Therapeutics Inc
Original Assignee
Amicus Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=65494618&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CN111971044(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CN111971044A publication Critical patent/CN111971044A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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

Landscapes

  • 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)
CN201980024425.7A 2018-02-06 2019-02-06 米加司他用于治疗妊娠患者的法布里病的用途 Pending CN111971044A (zh)

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
CN111971044A true CN111971044A (zh) 2020-11-20

Family

ID=65494618

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201980024425.7A Pending CN111971044A (zh) 2018-02-06 2019-02-06 米加司他用于治疗妊娠患者的法布里病的用途

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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101065667A (zh) * 2004-09-24 2007-10-31 貝丝以色列女执事医疗中心 诊断和治疗妊娠并发症的方法
CN103974619A (zh) * 2011-03-11 2014-08-06 阿米库斯医疗股份有限公司 治疗法布里病的给药方案
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 (22)

* 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
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
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
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

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101065667A (zh) * 2004-09-24 2007-10-31 貝丝以色列女执事医疗中心 诊断和治疗妊娠并发症的方法
CN103974619A (zh) * 2011-03-11 2014-08-06 阿米库斯医疗股份有限公司 治疗法布里病的给药方案
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
US11357784B2 (en) 2022-06-14
WO2019157047A1 (en) 2019-08-15
JP7555818B2 (ja) 2024-09-25
EP4717319A2 (en) 2026-04-01
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
KR20200128676A (ko) 2020-11-16
AU2024278298A1 (en) 2025-01-09
US20220387462A1 (en) 2022-12-08
EP3749307B1 (en) 2025-12-10

Similar Documents

Publication Publication Date Title
JP7784398B2 (ja) Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法
CN111971044A (zh) 米加司他用于治疗妊娠患者的法布里病的用途
AU2025217396A1 (en) Treatment of fabry disease in ert-naïve and ert-experienced patients
KR102631805B1 (ko) 신장 손상을 갖는 파브리 환자를 치료하는 방법
AU2018326364B2 (en) Methods of enhancing and/or stabilizing cardiac function in patients with Fabry disease
KR102004114B1 (ko) Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
AU2025200074A1 (en) Treatment of patients with classic fabry disease
JP2025131572A (ja) 腎機能障害を有する患者のファブリー病を治療する方法
JP2026016375A (ja) Ｇｌａ遺伝子の突然変異を有する患者におけるファブリー病を治療する方法
JP2025128105A (ja) ファブリー病患者の脳血管事象のリスク低減におけるミガロスタットの使用
CN111278438A (zh) 治疗具有肾损害的法布里患者的方法
HK40044013A (en) Use of migalastat for treating fabry disease in pregnant patients
HK40085360A (en) Methods of treating fabry disease in patients having a mutation in the gla gene
HK40039916A (en) Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
HK40044440A (en) Treatment of patients with classic fabry disease
HK40001720A (en) Methods of treating fabry disease in patients having the g9331a mutation in the gla gene
HK40031708A (en) Methods of treating fabry patients having renal impairment
HK40007037A (en) Treatment of fabry disease in ert-naïve and ert-experienced patients

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40044013

Country of ref document: HK