CA3110065A1 - Methods of treating fabry disease in patients having a mutation in the gla gene - Google Patents

Methods of treating fabry disease in patients having a mutation in the gla gene Download PDF

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Publication number
CA3110065A1
CA3110065A1 CA3110065A CA3110065A CA3110065A1 CA 3110065 A1 CA3110065 A1 CA 3110065A1 CA 3110065 A CA3110065 A CA 3110065A CA 3110065 A CA3110065 A CA 3110065A CA 3110065 A1 CA3110065 A1 CA 3110065A1
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CA
Canada
Prior art keywords
mutation
patient
migalastat
salt
administered
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
CA3110065A
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English (en)
French (fr)
Inventor
Elfrida Benjamin
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=65279726&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CA3110065(A1) "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 CA3110065A1 publication Critical patent/CA3110065A1/en
Pending legal-status Critical Current

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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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Cardiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Analytical Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Zoology (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pathology (AREA)
CA3110065A 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene Pending CA3110065A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862719962P 2018-08-20 2018-08-20
US62/719,962 2018-08-20
PCT/US2019/013761 WO2020040806A1 (en) 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene

Publications (1)

Publication Number Publication Date
CA3110065A1 true CA3110065A1 (en) 2020-02-27

Family

ID=65279726

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3110065A Pending CA3110065A1 (en) 2018-08-20 2019-01-16 Methods of treating fabry disease in patients having a mutation in the gla gene

Country Status (25)

Country Link
US (2) US12594268B2 (https=)
EP (7) EP4282472A3 (https=)
JP (3) JP2021535107A (https=)
KR (3) KR20240017127A (https=)
CN (1) CN112888439A (https=)
AU (2) AU2019326252A1 (https=)
BR (1) BR112021003137A2 (https=)
CA (1) CA3110065A1 (https=)
CL (3) CL2021000435A1 (https=)
DK (1) DK3840753T3 (https=)
EA (1) EA202190569A1 (https=)
ES (1) ES2993833T3 (https=)
FI (1) FI3840753T3 (https=)
HR (1) HRP20241454T1 (https=)
HU (1) HUE068783T2 (https=)
IL (2) IL325185A (https=)
LT (1) LT3840753T (https=)
MX (2) MX2021002070A (https=)
PL (1) PL3840753T3 (https=)
PT (1) PT3840753T (https=)
RS (1) RS66094B1 (https=)
SG (1) SG11202101117QA (https=)
SI (1) SI3840753T1 (https=)
SM (1) SMT202400436T1 (https=)
WO (1) WO2020040806A1 (https=)

Families Citing this family (10)

* 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
CN113969313A (zh) * 2021-11-29 2022-01-25 百世诺(北京)医学检验实验室有限公司 罕见遗传病突变基因及其应用
CN116622750B (zh) * 2023-06-01 2024-05-31 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途
CN116656704B (zh) * 2023-06-01 2024-11-05 华东理工大学 用于治疗法布里病的转基因表达盒

Family Cites Families (32)

* 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
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
EP2150254A4 (en) 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES 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
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
US20110070643A1 (en) 2009-05-26 2011-03-24 Do Hung V 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
US20120283290A1 (en) 2011-05-06 2012-11-08 Amicus Therapeutics Inc. Quantitation of gl3 in urine
CA2860085A1 (en) 2011-12-22 2013-06-27 Centogene Ip Gmbh Combination of a compound having the ability to rearrange a lysosomal enzyme and ambroxol and/or a derivative of ambroxol
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
WO2014120900A1 (en) * 2013-01-31 2014-08-07 Icahn School Of Medicine At Mount Sinai Enhanced therapeutic regimens for treating fabry disease
US20170051267A1 (en) 2015-08-20 2017-02-23 Research Foundation Of The City University Of New York Novel alpha-galactosidase a derivatives
KR102004114B1 (ko) * 2016-03-22 2019-07-26 아미쿠스 세라퓨틱스, 인코포레이티드 Ｇｌａ 유전자에 ｇ9331ａ 돌연변이를 갖는 환자에서 파브리 질병의 치료 방법
AR109103A1 (es) 2016-07-19 2018-10-31 Amicus Therapeutics Inc Tratamiento de la enfermedad de fabry en pacientes no tratados y tratados previamente con ert
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
WO2019017938A1 (en) 2017-07-19 2019-01-24 Amicus Therapeutics, Inc. TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES
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
TW202042812A (zh) * 2019-01-22 2020-12-01 美商阿米庫斯醫療股份有限公司 減少法布瑞氏症患者中之腦血管事件之方法
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
EP4114390B1 (en) * 2020-03-06 2024-12-04 Amicus Therapeutics, Inc. Methods of treating fabry disease in patients having a mutation in the gla gene

Also Published As

Publication number Publication date
IL325185A (en) 2026-02-01
CL2024000741A1 (es) 2024-10-04
WO2020040806A1 (en) 2020-02-27
EP3840753A1 (en) 2021-06-30
KR20210046729A (ko) 2021-04-28
ES2993833T3 (en) 2025-01-10
EP4289475A2 (en) 2023-12-13
EP4289476A3 (en) 2024-02-21
AU2019326252A1 (en) 2021-02-25
AU2025205141A1 (en) 2025-07-31
IL280961B1 (en) 2026-01-01
BR112021003137A2 (pt) 2021-05-11
PT3840753T (pt) 2024-10-30
EP4289476A2 (en) 2023-12-13
HUE068783T2 (hu) 2025-01-28
JP2021535107A (ja) 2021-12-16
EP4285990A3 (en) 2024-02-21
EP3840753B1 (en) 2024-07-24
EP4282472A3 (en) 2024-02-21
CL2024000739A1 (es) 2024-10-04
SG11202101117QA (en) 2021-03-30
EP4299117A2 (en) 2024-01-03
US20240277687A1 (en) 2024-08-22
JP2023106398A (ja) 2023-08-01
KR20240017127A (ko) 2024-02-06
US20210315875A1 (en) 2021-10-14
DK3840753T3 (da) 2024-10-28
EP4285991A2 (en) 2023-12-06
EP4289475A3 (en) 2024-02-21
MX2021002070A (es) 2021-07-15
MX2024008350A (es) 2024-07-19
EA202190569A1 (ru) 2021-06-01
SI3840753T1 (sl) 2025-01-31
FI3840753T3 (fi) 2024-10-30
RS66094B1 (sr) 2024-11-29
SMT202400436T1 (it) 2024-11-15
CN112888439A (zh) 2021-06-01
HRP20241454T1 (hr) 2024-12-20
JP2025165955A (ja) 2025-11-05
CL2021000435A1 (es) 2021-10-22
JP7784398B2 (ja) 2025-12-11
EP4282472A2 (en) 2023-11-29
IL280961A (en) 2021-04-29
EP4299117A3 (en) 2024-02-21
KR20240017128A (ko) 2024-02-06
PL3840753T3 (pl) 2025-01-20
EP4285990A2 (en) 2023-12-06
EP4285991A3 (en) 2024-02-21
US12594268B2 (en) 2026-04-07
LT3840753T (lt) 2024-11-11

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