FI4176877T3 - Menetelmiä sydämen toiminnan tehostamiseksi ja/tai stabiloimiseksi potilaissa, joilla on fabryn tauti - Google Patents
Menetelmiä sydämen toiminnan tehostamiseksi ja/tai stabiloimiseksi potilaissa, joilla on fabryn tauti Download PDFInfo
- Publication number
- FI4176877T3 FI4176877T3 FIEP22194251.9T FI22194251T FI4176877T3 FI 4176877 T3 FI4176877 T3 FI 4176877T3 FI 22194251 T FI22194251 T FI 22194251T FI 4176877 T3 FI4176877 T3 FI 4176877T3
- Authority
- FI
- Finland
- Prior art keywords
- migalastat
- salt
- use according
- patient
- administered
- Prior art date
Links
- 208000024720 Fabry Disease Diseases 0.000 title claims 2
- 230000004217 heart function Effects 0.000 title claims 2
- 229950007469 migalastat Drugs 0.000 claims 15
- LXBIFEVIBLOUGU-DPYQTVNSSA-N migalastat Chemical compound OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O LXBIFEVIBLOUGU-DPYQTVNSSA-N 0.000 claims 15
- 150000003839 salts Chemical class 0.000 claims 15
- 238000009472 formulation Methods 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 239000012458 free base Substances 0.000 claims 2
- 239000006186 oral dosage form Substances 0.000 claims 2
- ZJIHMALTJRDNQI-OLALXQGDSA-N (2r,3s,4r,5s)-2-(hydroxymethyl)piperidine-3,4,5-triol;hydrochloride Chemical compound Cl.OC[C@H]1NC[C@H](O)[C@@H](O)[C@H]1O ZJIHMALTJRDNQI-OLALXQGDSA-N 0.000 claims 1
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 1
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 1
- 238000003556 assay Methods 0.000 claims 1
- 239000002775 capsule Substances 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 238000002641 enzyme replacement therapy Methods 0.000 claims 1
- 230000001771 impaired effect Effects 0.000 claims 1
- 230000000977 initiatory effect Effects 0.000 claims 1
- 229950010505 migalastat hydrochloride Drugs 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 238000004904 shortening Methods 0.000 claims 1
- 230000000087 stabilizing effect Effects 0.000 claims 1
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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (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 (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762550984P | 2017-08-28 | 2017-08-28 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| FI4176877T3 true FI4176877T3 (fi) | 2025-03-06 |
Family
ID=63528948
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| FIEP22194251.9T FI4176877T3 (fi) | 2017-08-28 | 2018-08-28 | Menetelmiä sydämen toiminnan tehostamiseksi ja/tai stabiloimiseksi potilaissa, joilla on fabryn tauti |
Country Status (26)
| Country | Link |
|---|---|
| US (1) | US20200222377A1 (enExample) |
| EP (3) | EP3675853B1 (enExample) |
| JP (3) | JP7755929B2 (enExample) |
| KR (2) | KR102790184B1 (enExample) |
| CN (1) | CN111770753A (enExample) |
| AR (1) | AR112482A1 (enExample) |
| AU (2) | AU2018326364B2 (enExample) |
| CA (1) | CA3074450C (enExample) |
| CL (1) | CL2020000491A1 (enExample) |
| DK (2) | DK4176877T3 (enExample) |
| EA (1) | EA202090564A1 (enExample) |
| ES (2) | ES2931054T3 (enExample) |
| FI (1) | FI4176877T3 (enExample) |
| HR (2) | HRP20250064T1 (enExample) |
| HU (2) | HUE070433T2 (enExample) |
| IL (3) | IL272893B2 (enExample) |
| LT (2) | LT4176877T (enExample) |
| MX (2) | MX2020002229A (enExample) |
| PL (2) | PL4176877T3 (enExample) |
| PT (2) | PT4176877T (enExample) |
| RS (2) | RS66515B1 (enExample) |
| SG (1) | SG11202001707PA (enExample) |
| SI (1) | SI4176877T1 (enExample) |
| SM (2) | SMT202500073T1 (enExample) |
| TW (1) | TWI845479B (enExample) |
| WO (1) | WO2019046244A1 (enExample) |
Families Citing this family (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| AU2018277756A1 (en) | 2017-05-30 | 2020-01-23 | Amicus Therapeutics, Inc. | Methods of treating fabry patients having renal impairment |
| WO2019046244A1 (en) | 2017-08-28 | 2019-03-07 | Amicus Therapeutics, Inc. | METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE |
| EP4717319A3 (en) | 2018-02-06 | 2026-06-10 | Amicus Therapeutics, Inc. | Methods of treating fabry disease in pregnant patients |
| LT3840753T (lt) | 2018-08-20 | 2024-11-11 | Amicus Therapeutics, Inc. | Migalastatas skirtas naudoti fabry ligos gydymo būdui, pacientams turintiems gla geno mutaciją |
| 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 |
| TR2024011149A1 (tr) | 2024-08-23 | 2026-03-23 | Sanovel Ilac Sanayi Ve Ticaret Anonim Sirketi | 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 |
Family Cites Families (6)
| 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 |
| EP2142197A4 (en) | 2007-03-30 | 2010-11-10 | Amicus Therapeutics Inc | METHOD FOR THE TREATMENT OF MORBUS FABRY 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 |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| WO2019046244A1 (en) | 2017-08-28 | 2019-03-07 | Amicus Therapeutics, Inc. | METHODS FOR ENHANCING AND / OR STABILIZING CARDIAC FUNCTION IN PATIENTS WITH FABRY DISEASE |
| US10811814B2 (en) | 2017-08-28 | 2020-10-20 | John Mezzalingua Associates, LLC | Weather protecting (WP) housing for coaxial cable connectors |
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2018
- 2018-08-28 WO PCT/US2018/048257 patent/WO2019046244A1/en not_active Ceased
- 2018-08-28 MX MX2020002229A patent/MX2020002229A/es unknown
- 2018-08-28 AU AU2018326364A patent/AU2018326364B2/en active Active
- 2018-08-28 PL PL22194251.9T patent/PL4176877T3/pl 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 HU HUE22194251A patent/HUE070433T2/hu unknown
- 2018-08-28 SM SM20250073T patent/SMT202500073T1/it unknown
- 2018-08-28 PT PT221942519T patent/PT4176877T/pt unknown
- 2018-08-28 IL IL272893A patent/IL272893B2/en unknown
- 2018-08-28 AR ARP180102438 patent/AR112482A1/es unknown
- 2018-08-28 LT LTEP22194251.9T patent/LT4176877T/lt unknown
- 2018-08-28 JP JP2020511971A patent/JP7755929B2/ja active Active
- 2018-08-28 SI SI201831201T patent/SI4176877T1/sl unknown
- 2018-08-28 PL PL18766482.6T patent/PL3675853T3/pl unknown
- 2018-08-28 RS RS20250161A patent/RS66515B1/sr unknown
- 2018-08-28 HU HUE18766482A patent/HUE060287T2/hu unknown
- 2018-08-28 EP EP18766482.6A patent/EP3675853B1/en active Active
- 2018-08-28 HR HRP20250064TT patent/HRP20250064T1/hr unknown
- 2018-08-28 HR HRP20221366TT patent/HRP20221366T1/hr unknown
- 2018-08-28 SG SG11202001707PA patent/SG11202001707PA/en unknown
- 2018-08-28 IL IL310648A patent/IL310648A/en unknown
- 2018-08-28 FI FIEP22194251.9T patent/FI4176877T3/fi active
- 2018-08-28 ES ES18766482T patent/ES2931054T3/es active Active
- 2018-08-28 PT PT187664826T patent/PT3675853T/pt unknown
- 2018-08-28 LT LTEPPCT/US2018/048257T patent/LT3675853T/lt unknown
- 2018-08-28 EP EP24217100.7A patent/EP4588476A1/en active Pending
- 2018-08-28 EA EA202090564A patent/EA202090564A1/ru unknown
- 2018-08-28 SM SM20220459T patent/SMT202200459T1/it unknown
- 2018-08-28 KR KR1020207008736A patent/KR102790184B1/ko active Active
- 2018-08-28 DK DK18766482.6T patent/DK3675853T3/da active
- 2018-08-28 RS RS20221061A patent/RS63742B1/sr unknown
- 2018-08-28 ES ES22194251T patent/ES3013820T3/es active Active
- 2018-08-28 EP EP22194251.9A patent/EP4176877B1/en active Active
- 2018-08-28 CA CA3074450A patent/CA3074450C/en active Active
- 2018-08-28 KR KR1020257010249A patent/KR20250049561A/ko active Pending
- 2018-08-28 CN CN201880056413.8A patent/CN111770753A/zh active Pending
- 2018-08-28 TW TW107129901A patent/TWI845479B/zh active
-
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
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