IL272893B2 - Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease - Google Patents
Methods of enhancing and/or stabilizing cardiac function in patients with fabry diseaseInfo
- Publication number
- IL272893B2 IL272893B2 IL272893A IL27289320A IL272893B2 IL 272893 B2 IL272893 B2 IL 272893B2 IL 272893 A IL272893 A IL 272893A IL 27289320 A IL27289320 A IL 27289320A IL 272893 B2 IL272893 B2 IL 272893B2
- Authority
- IL
- Israel
- Prior art keywords
- formulation
- migalastat
- patient
- salt
- mwfs
- Prior art date
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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762550984P | 2017-08-28 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| IL272893A IL272893A (en) | 2020-04-30 |
| IL272893B1 IL272893B1 (en) | 2024-03-01 |
| IL272893B2 true IL272893B2 (en) | 2024-07-01 |
Family
ID=63528948
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL272893A IL272893B2 (en) | 2017-08-28 | 2018-08-28 | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease |
| IL310648A IL310648A (en) | 2017-08-28 | 2018-08-28 | Methods for increasing and/or stabilizing cardiac functions in patients with Fabry disease |
| IL324182A IL324182A (en) | 2017-08-28 | 2025-10-23 | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL310648A IL310648A (en) | 2017-08-28 | 2018-08-28 | Methods for increasing and/or stabilizing cardiac functions in patients with Fabry disease |
| IL324182A IL324182A (en) | 2017-08-28 | 2025-10-23 | Methods of enhancing and/or stabilizing cardiac function in patients with fabry disease |
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)
| 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)
| 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)
| 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 |
-
2018
- 2018-08-28 IL IL272893A patent/IL272893B2/en unknown
- 2018-08-28 SI SI201831201T patent/SI4176877T1/sl unknown
- 2018-08-28 DK DK18766482.6T patent/DK3675853T3/da active
- 2018-08-28 MX MX2020002229A patent/MX2020002229A/es unknown
- 2018-08-28 FI FIEP22194251.9T patent/FI4176877T3/fi active
- 2018-08-28 TW TW107129901A patent/TWI845479B/zh active
- 2018-08-28 KR KR1020207008736A patent/KR102790184B1/ko active Active
- 2018-08-28 PL PL22194251.9T patent/PL4176877T3/pl unknown
- 2018-08-28 EP EP24217100.7A patent/EP4588476A1/en active Pending
- 2018-08-28 CN CN201880056413.8A patent/CN111770753A/zh active Pending
- 2018-08-28 RS RS20250161A patent/RS66515B1/sr unknown
- 2018-08-28 IL IL310648A patent/IL310648A/en unknown
- 2018-08-28 PT PT221942519T patent/PT4176877T/pt unknown
- 2018-08-28 EP EP18766482.6A patent/EP3675853B1/en active Active
- 2018-08-28 RS RS20221061A patent/RS63742B1/sr unknown
- 2018-08-28 AU AU2018326364A patent/AU2018326364B2/en active Active
- 2018-08-28 LT LTEP22194251.9T patent/LT4176877T/lt unknown
- 2018-08-28 SG SG11202001707PA patent/SG11202001707PA/en unknown
- 2018-08-28 JP JP2020511971A patent/JP7755929B2/ja active Active
- 2018-08-28 SM SM20220459T patent/SMT202200459T1/it unknown
- 2018-08-28 US US16/642,620 patent/US20200222377A1/en active Pending
- 2018-08-28 DK DK22194251.9T patent/DK4176877T3/da active
- 2018-08-28 PL PL18766482.6T patent/PL3675853T3/pl unknown
- 2018-08-28 WO PCT/US2018/048257 patent/WO2019046244A1/en not_active Ceased
- 2018-08-28 HR HRP20250064TT patent/HRP20250064T1/hr unknown
- 2018-08-28 EP EP22194251.9A patent/EP4176877B1/en active Active
- 2018-08-28 HU HUE18766482A patent/HUE060287T2/hu unknown
- 2018-08-28 ES ES18766482T patent/ES2931054T3/es active Active
- 2018-08-28 HU HUE22194251A patent/HUE070433T2/hu unknown
- 2018-08-28 AR ARP180102438 patent/AR112482A1/es unknown
- 2018-08-28 LT LTEPPCT/US2018/048257T patent/LT3675853T/lt unknown
- 2018-08-28 SM SM20250073T patent/SMT202500073T1/it unknown
- 2018-08-28 PT PT187664826T patent/PT3675853T/pt unknown
- 2018-08-28 KR KR1020257010249A patent/KR20250049561A/ko active Pending
- 2018-08-28 HR HRP20221366TT patent/HRP20221366T1/hr unknown
- 2018-08-28 CA CA3074450A patent/CA3074450C/en active Active
- 2018-08-28 ES ES22194251T patent/ES3013820T3/es active Active
- 2018-08-28 EA EA202090564A patent/EA202090564A1/ru unknown
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2020
- 2020-02-27 MX MX2023005628A patent/MX2023005628A/es unknown
- 2020-02-28 CL CL2020000491A patent/CL2020000491A1/es unknown
-
2022
- 2022-12-20 JP JP2022203783A patent/JP2023051998A/ja active Pending
-
2024
- 2024-11-27 AU AU2024266945A patent/AU2024266945A1/en active Pending
-
2025
- 2025-05-28 JP JP2025088523A patent/JP2025138638A/ja active Pending
- 2025-10-23 IL IL324182A patent/IL324182A/en unknown
Patent Citations (1)
| 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)
| Title |
|---|
| DOMINIQUE P. GERMAIN ET AL,, TREATMENT OF FABRY'S DISEASE WITH THE PHARMACOLOGIC CHAPERONE MIGALASTAT, 11 August 2016 (2016-08-11) * |
| GOKER-ALPAN O ET AL, " EFFECT AND TOLERABILITY OF AGALSIDASE ALFA IN PATIENTS WITH FABRY DISEASE WHO WERE TREATMENT NA?VE OR FORMERLY TREATED WITH AGALSIDASE BETA OR AGALSIDASE ALFA", 30 March 2015 (2015-03-30) * |
| GOKER-ALPAN O ET.AL, " AN OPEN-LABEL CLINICAL TRIAL OF AGALSIDASE ALFA ENZYME REPLACEMENT THERAPY IN CHILDREN WITH FABRY DISEASE WHO ARE NA?VE TO ENZYME REPLACEMENT THERAPY", 18 December 2015 (2015-12-18) * |
| HUGHES DA, ET AL., " ORAL PHARMACOLOGICAL CHAPERONE MIGALASTAT COMPARED WITH ENZYME REPLACEMENT THERAPY IN FABRY DISEASE: 18-MONTH RESULTS FROM THE RANDOMISED PHASE III ATTRACT STUDY", 10 November 2016 (2016-11-10) * |
| MEHTA A. ET AL ON BEHALF OF THE FABRY OUTCOME SURVEY INVESTIGATORS,, ENZYME REPLACEMENT THERAPY WITH AGALSIDASE ALFA IN PATIENTS WITH FABRY'S DISEASE: AN ANALYSIS OF REGISTRY DATA, 12 December 2009 (2009-12-12) * |
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