JP7755929B2 - ファブリー病患者における心臓機能の強化及び／または安定化方法 - Google Patents

ファブリー病患者における心臓機能の強化及び／または安定化方法

Info

Publication number
JP7755929B2
JP7755929B2 JP2020511971A JP2020511971A JP7755929B2 JP 7755929 B2 JP7755929 B2 JP 7755929B2 JP 2020511971 A JP2020511971 A JP 2020511971A JP 2020511971 A JP2020511971 A JP 2020511971A JP 7755929 B2 JP7755929 B2 JP 7755929B2
Authority
JP
Japan
Prior art keywords
migalastat
patient
salt
mwfs
patients
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.)
Active
Application number
JP2020511971A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020531550A5 (https=
JPWO2019046244A5 (https=
JP2020531550A (ja
Inventor
ジェフ カステリ，
ジェイ バース，
ニーナ スクバン，
Original Assignee
アミカス セラピューティックス インコーポレイテッド
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=63528948&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP7755929(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by アミカス セラピューティックス インコーポレイテッド filed Critical アミカス セラピューティックス インコーポレイテッド
Publication of JP2020531550A publication Critical patent/JP2020531550A/ja
Priority to JP2022203783A priority Critical patent/JP2023051998A/ja
Priority to JP2025088523A priority patent/JP2025138638A/ja
Publication of JP2020531550A5 publication Critical patent/JP2020531550A5/ja
Publication of JPWO2019046244A5 publication Critical patent/JPWO2019046244A5/ja
Application granted granted Critical
Publication of JP7755929B2 publication Critical patent/JP7755929B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

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
    • 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
    • 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
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • A61K9/08—Solutions
    • 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
    • A61K9/20—Pills, tablets, discs, rods
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cardiology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2020511971A 2017-08-28 2018-08-28 ファブリー病患者における心臓機能の強化及び／または安定化方法 Active JP7755929B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2022203783A JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762550984P 2017-08-28 2017-08-28
US62/550,984 2017-08-28
PCT/US2018/048257 WO2019046244A1 (en) 2017-08-28 2018-08-28 METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2022203783A Division JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A Division JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Publications (4)

Publication Number Publication Date
JP2020531550A JP2020531550A (ja) 2020-11-05
JP2020531550A5 JP2020531550A5 (https=) 2025-06-10
JPWO2019046244A5 JPWO2019046244A5 (https=) 2025-06-10
JP7755929B2 true JP7755929B2 (ja) 2025-10-17

Family

ID=63528948

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2020511971A Active JP7755929B2 (ja) 2017-08-28 2018-08-28 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2022203783A Pending JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A Pending JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2022203783A Pending JP2023051998A (ja) 2017-08-28 2022-12-20 ファブリー病患者における心臓機能の強化及び／または安定化方法
JP2025088523A Pending JP2025138638A (ja) 2017-08-28 2025-05-28 ファブリー病患者における心臓機能の強化及び／または安定化方法

Country Status (26)

Country Link
US (1) US20200222377A1 (https=)
EP (3) EP4588476A1 (https=)
JP (3) JP7755929B2 (https=)
KR (2) KR102790184B1 (https=)
CN (1) CN111770753A (https=)
AR (1) AR112482A1 (https=)
AU (2) AU2018326364B2 (https=)
CA (1) CA3074450C (https=)
CL (1) CL2020000491A1 (https=)
DK (2) DK3675853T3 (https=)
EA (1) EA202090564A1 (https=)
ES (2) ES2931054T3 (https=)
FI (1) FI4176877T3 (https=)
HR (2) HRP20250064T1 (https=)
HU (2) HUE060287T2 (https=)
IL (3) IL272893B2 (https=)
LT (2) LT4176877T (https=)
MX (2) MX2020002229A (https=)
PL (2) PL4176877T3 (https=)
PT (2) PT4176877T (https=)
RS (2) RS66515B1 (https=)
SG (1) SG11202001707PA (https=)
SI (1) SI4176877T1 (https=)
SM (2) SMT202200459T1 (https=)
TW (1) TWI845479B (https=)
WO (1) WO2019046244A1 (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
IL272893B2 (en) 2017-08-28 2024-07-01 Amicus Therapeutics Inc Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
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
TR2024011149A1 (tr) 2024-08-23 2026-03-23 Sanovel İlaç Sanayi̇ Ve Ti̇caret Anoni̇m Şi̇rketi̇ Mi̇galastat i̇çeren kapsül formülasyonlari
CN119120696A (zh) * 2024-11-12 2024-12-13 北京贝瑞和康生物技术有限公司 检测法布雷病gla基因突变的引物组、试剂盒及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160324839A1 (en) 2007-04-26 2016-11-10 Amicus Therapeutics, Inc. Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones

Family Cites Families (5)

* 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
AU2008232614A1 (en) 2007-03-30 2008-10-09 Amicus Therapeutics, Inc. Method for the treatment of Fabry disease using pharmacological chaperones
SI3470077T1 (sl) 2008-02-12 2020-12-31 Amicus Therapeutics, Inc. Postopek za napovedovanje odgovora na zdravljenje bolezni s farmakološkim spremljevalcem
IL272893B2 (en) 2017-08-28 2024-07-01 Amicus Therapeutics Inc Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
CN111344912B (zh) 2017-08-28 2021-12-28 约翰·梅扎林瓜联合股份有限公司 同轴电缆连接器的天气防护壳体

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20160324839A1 (en) 2007-04-26 2016-11-10 Amicus Therapeutics, Inc. Dosing Regimens for the Treatment of Lysosomal Storage Diseases Using Pharmacological Chaperones

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
Drug Design, Development and Therapy, 2016年, 発行日, Vol.10, pp.1771-1781
J Med Genet, 2017年, 発行日, Vol.54, pp.288-298
JIMD Reports, 2015年, 発行日, Vol.23, pp.7-15
Lancet, 2009年, 発行日, Vol.374, pp.1986-1996
N Engl J Med, 2016年, 発行日, Vol.375, pp.545-555

Also Published As

Publication number Publication date
SMT202200459T1 (it) 2023-01-13
JP2025138638A (ja) 2025-09-25
MX2020002229A (es) 2020-07-20
TWI845479B (zh) 2024-06-21
RS63742B1 (sr) 2022-12-30
SG11202001707PA (en) 2020-03-30
PT3675853T (pt) 2022-11-28
HRP20250064T1 (hr) 2025-03-14
KR20250049561A (ko) 2025-04-11
IL324182A (en) 2025-12-01
EA202090564A1 (ru) 2020-06-15
EP3675853A1 (en) 2020-07-08
KR102790184B1 (ko) 2025-04-01
RS66515B1 (sr) 2025-03-31
IL272893B2 (en) 2024-07-01
IL310648A (en) 2024-04-01
SI4176877T1 (sl) 2025-03-31
AR112482A1 (es) 2019-10-30
EP3675853B1 (en) 2022-09-07
US20200222377A1 (en) 2020-07-16
AU2018326364B2 (en) 2024-08-29
IL272893A (en) 2020-04-30
CL2020000491A1 (es) 2020-09-11
IL272893B1 (en) 2024-03-01
DK4176877T3 (da) 2025-02-24
EP4176877B1 (en) 2024-12-04
MX2023005628A (es) 2023-05-24
SMT202500073T1 (it) 2025-03-12
LT3675853T (lt) 2022-12-12
EP4588476A1 (en) 2025-07-23
HUE060287T2 (hu) 2023-02-28
LT4176877T (lt) 2025-02-10
HRP20221366T1 (hr) 2023-01-06
TW201919619A (zh) 2019-06-01
ES2931054T3 (es) 2022-12-23
PL3675853T3 (pl) 2022-12-27
JP2020531550A (ja) 2020-11-05
AU2018326364A1 (en) 2020-04-09
DK3675853T3 (da) 2022-11-28
BR112020003974A2 (pt) 2020-09-01
ES3013820T3 (en) 2025-04-15
PT4176877T (pt) 2025-02-24
PL4176877T3 (pl) 2025-04-07
AU2024266945A1 (en) 2024-12-19
FI4176877T3 (fi) 2025-03-06
CN111770753A (zh) 2020-10-13
WO2019046244A1 (en) 2019-03-07
JP2023051998A (ja) 2023-04-11
CA3074450C (en) 2024-10-01
CA3074450A1 (en) 2019-03-07
KR20200044908A (ko) 2020-04-29
HUE070433T2 (hu) 2025-06-28
EP4176877A1 (en) 2023-05-10

Similar Documents

Publication Publication Date Title
JP7755929B2 (ja) ファブリー病患者における心臓機能の強化及び／または安定化方法
US12599594B2 (en) Treatment of Fabry disease in ERT-naïve and ERT-experienced patients
TWI869336B (zh) 經典型Fabry氏病患者之治療
IL280961B1 (en) Methods for treating Fabry disease in patients with a mutation in the gla gene
JP2023513700A (ja) ファブリー病の治療方法
WO2019017938A1 (en) TREATMENT OF FABRY'S DISEASE IN PATIENTS WITHOUT SUBSTITUTED ENZYMATIC THERAPY (TES) AND PATIENTS SUBJECT TO TES
JP7749572B2 (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
JP7680356B2 (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
JP2024525760A (ja) 小児患者のファブリー病を治療する方法
HK40128496A (en) Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease
HK40106025A (en) Treatment of patients with classic fabry disease
EA045263B1 (ru) Способы усиления и/или стабилизации сердечной функции у пациентов с болезнью фабри
BR112020003974B1 (pt) Métodos para intensificar e/ou estabilizar função cardíaca em pacientes com doença de fabry

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210827

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210827

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220621

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20220622

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220920

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20221121

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20230228

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20250502

A524 Written submission of copy of amendment under article 19 pct

Free format text: JAPANESE INTERMEDIATE CODE: A524

Effective date: 20250528

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20251006

R150 Certificate of patent or registration of utility model

Ref document number: 7755929

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150