CN112888439A - 治疗在gla基因中具有突变的患者的法布里病的方法 - Google Patents
治疗在gla基因中具有突变的患者的法布里病的方法 Download PDFInfo
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- CN112888439A CN112888439A CN201980069272.8A CN201980069272A CN112888439A CN 112888439 A CN112888439 A CN 112888439A CN 201980069272 A CN201980069272 A CN 201980069272A CN 112888439 A CN112888439 A CN 112888439A
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- 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
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- 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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- Life Sciences & Earth Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
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- General Chemical & Material Sciences (AREA)
- Epidemiology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
- Urology & Nephrology (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)
- Analytical Chemistry (AREA)
- Molecular Biology (AREA)
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- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Biophysics (AREA)
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- Biotechnology (AREA)
- Immunology (AREA)
- Microbiology (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Pathology (AREA)
- Biochemistry (AREA)
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 |
|---|---|
| CN112888439A true CN112888439A (zh) | 2021-06-01 |
Family
ID=65279726
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201980069272.8A Pending CN112888439A (zh) | 2018-08-20 | 2019-01-16 | 治疗在gla基因中具有突变的患者的法布里病的方法 |
Country Status (25)
| Country | Link |
|---|---|
| US (2) | US12594268B2 (enExample) |
| EP (7) | EP3840753B1 (enExample) |
| JP (3) | JP2021535107A (enExample) |
| KR (3) | KR20240017127A (enExample) |
| CN (1) | CN112888439A (enExample) |
| AU (2) | AU2019326252A1 (enExample) |
| BR (1) | BR112021003137A2 (enExample) |
| CA (1) | CA3110065A1 (enExample) |
| CL (3) | CL2021000435A1 (enExample) |
| DK (1) | DK3840753T3 (enExample) |
| EA (1) | EA202190569A1 (enExample) |
| ES (1) | ES2993833T3 (enExample) |
| FI (1) | FI3840753T3 (enExample) |
| HR (1) | HRP20241454T1 (enExample) |
| HU (1) | HUE068783T2 (enExample) |
| IL (2) | IL325185A (enExample) |
| LT (1) | LT3840753T (enExample) |
| MX (2) | MX2021002070A (enExample) |
| PL (1) | PL3840753T3 (enExample) |
| PT (1) | PT3840753T (enExample) |
| RS (1) | RS66094B1 (enExample) |
| SG (1) | SG11202101117QA (enExample) |
| SI (1) | SI3840753T1 (enExample) |
| SM (1) | SMT202400436T1 (enExample) |
| WO (1) | WO2020040806A1 (enExample) |
Cited By (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CN116622750A (zh) * | 2023-06-01 | 2023-08-22 | 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 |
| CN116656704A (zh) * | 2023-06-01 | 2023-08-29 | 华东理工大学 | 用于治疗法布里病的转基因表达盒 |
Families Citing this family (7)
| 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 (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110152319A1 (en) * | 2008-02-12 | 2011-06-23 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| 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 |
| WO2014120900A1 (en) * | 2013-01-31 | 2014-08-07 | Icahn School Of Medicine At Mount Sinai | Enhanced therapeutic regimens for treating fabry disease |
| TW201733581A (zh) * | 2016-03-22 | 2017-10-01 | 阿米庫斯醫療股份有限公司 | 在gla基因中具有g9331a突變的患者中治療法布瑞氏症的方法 |
| WO2018017721A1 (en) * | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| WO2018132471A1 (en) * | 2017-01-10 | 2018-07-19 | Amicus Therapeutics, Inc. | Recombinant alpha-galactosidase a for treatment of fabry disease |
Family Cites Families (26)
| 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 |
| WO2010048532A1 (en) | 2008-10-24 | 2010-04-29 | Amicus Therapeutics, Inc. | Multiple compartment dosing model |
| AU2010254092B2 (en) | 2009-05-26 | 2015-11-12 | Amicus Therapeutics, Inc. | Utilization of pharmacological chaperones to improve manufacturing and purification of biologics |
| 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 |
| US20120283290A1 (en) | 2011-05-06 | 2012-11-08 | Amicus Therapeutics Inc. | Quantitation of gl3 in urine |
| KR102076585B1 (ko) | 2011-12-22 | 2020-02-12 | 센토제너 아이피 게엠베하 | 리소좀 효소를 재배열하는 능력을 갖는 화합물 및 암브록솔 및/또는 암브록솔의 유도체의 조합물 |
| 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 |
| US20170051267A1 (en) | 2015-08-20 | 2017-02-23 | Research Foundation Of The City University Of New York | Novel alpha-galactosidase a derivatives |
| CA3224546C (en) | 2017-05-30 | 2026-02-24 | Amicus Therapeutics, Inc. | Use of migalastat in the treatment of fabry patients |
| SG11202000423XA (en) | 2017-07-19 | 2020-02-27 | Amicus Therapeutics Inc | Treatment of fabry disease in ert-naïve and ert-experienced 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 |
| 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 |
| 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 |
| AU2021230371A1 (en) * | 2020-03-06 | 2022-09-29 | Amicus Therapeutics, Inc. | Methods of treating Fabry disease in patients having a mutation in the GLA gene |
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2019
- 2019-01-16 US US17/269,890 patent/US12594268B2/en active Active
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- 2019-01-16 IL IL325185A patent/IL325185A/en unknown
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- 2019-01-16 DK DK19703514.0T patent/DK3840753T3/da active
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- 2019-01-16 PL PL19703514.0T patent/PL3840753T3/pl unknown
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- 2019-01-16 BR BR112021003137-5A patent/BR112021003137A2/pt unknown
- 2019-01-16 HU HUE19703514A patent/HUE068783T2/hu unknown
- 2019-01-16 FI FIEP19703514.0T patent/FI3840753T3/fi active
- 2019-01-16 KR KR1020247003292A patent/KR20240017128A/ko active Pending
- 2019-01-16 WO PCT/US2019/013761 patent/WO2020040806A1/en not_active Ceased
- 2019-01-16 SM SM20240436T patent/SMT202400436T1/it unknown
- 2019-01-16 SG SG11202101117QA patent/SG11202101117QA/en unknown
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- 2019-01-16 PT PT197035140T patent/PT3840753T/pt unknown
- 2019-01-16 HR HRP20241454TT patent/HRP20241454T1/hr unknown
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- 2019-01-16 EP EP23202198.0A patent/EP4289476A3/en not_active Withdrawn
- 2019-01-16 RS RS20241181A patent/RS66094B1/sr unknown
- 2019-01-16 EP EP23202201.2A patent/EP4285991A3/en not_active Withdrawn
- 2019-01-16 SI SI201930852T patent/SI3840753T1/sl unknown
- 2019-01-16 MX MX2021002070A patent/MX2021002070A/es unknown
- 2019-01-16 EP EP23202183.2A patent/EP4289475A3/en active Pending
- 2019-01-16 ES ES19703514T patent/ES2993833T3/es active Active
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2021
- 2021-02-18 IL IL280961A patent/IL280961B1/en unknown
- 2021-02-19 MX MX2024008350A patent/MX2024008350A/es unknown
- 2021-02-19 CL CL2021000435A patent/CL2021000435A1/es unknown
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2023
- 2023-04-25 JP JP2023071652A patent/JP7784398B2/ja active Active
- 2023-10-02 US US18/479,396 patent/US20240277687A1/en active Pending
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2024
- 2024-03-13 CL CL2024000741A patent/CL2024000741A1/es unknown
- 2024-03-13 CL CL2024000739A patent/CL2024000739A1/es unknown
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2025
- 2025-07-04 AU AU2025205141A patent/AU2025205141A1/en active Pending
- 2025-07-14 JP JP2025117816A patent/JP2025165955A/ja active Pending
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20110152319A1 (en) * | 2008-02-12 | 2011-06-23 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| 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 |
| WO2014120900A1 (en) * | 2013-01-31 | 2014-08-07 | Icahn School Of Medicine At Mount Sinai | Enhanced therapeutic regimens for treating fabry disease |
| TW201733581A (zh) * | 2016-03-22 | 2017-10-01 | 阿米庫斯醫療股份有限公司 | 在gla基因中具有g9331a突變的患者中治療法布瑞氏症的方法 |
| WO2018017721A1 (en) * | 2016-07-19 | 2018-01-25 | Amicus Therapeutics, Inc. | Treatment of fabry disease in ert-naïve and ert-experienced patients |
| WO2018132471A1 (en) * | 2017-01-10 | 2018-07-19 | Amicus Therapeutics, Inc. | Recombinant alpha-galactosidase a for treatment of fabry disease |
Non-Patent Citations (3)
| Title |
|---|
| ELFRIDA R. BENJAMIN等: "The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat", GENET MED., vol. 19, no. 4, pages 7 * |
| 佚名: "Drug Trials Snapshots_ GALAFOLD _ FDA", Retrieved from the Internet <URL:https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-galafold> * |
| 杨君义;: "治疗法布里病新药――migalastat", 中国新药与临床杂志, no. 05 * |
Cited By (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| CN116622750A (zh) * | 2023-06-01 | 2023-08-22 | 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 |
| CN116656704A (zh) * | 2023-06-01 | 2023-08-29 | 华东理工大学 | 用于治疗法布里病的转基因表达盒 |
| 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 |
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