DK3840753T3 - Migalastat til anvendelse i fremgangsmåder til behandling af fabrys sygdom hos patienter, der har en mutation i gla-genet - Google Patents
Migalastat til anvendelse i fremgangsmåder til behandling af fabrys sygdom hos patienter, der har en mutation i gla-genet Download PDFInfo
- Publication number
- DK3840753T3 DK3840753T3 DK19703514.0T DK19703514T DK3840753T3 DK 3840753 T3 DK3840753 T3 DK 3840753T3 DK 19703514 T DK19703514 T DK 19703514T DK 3840753 T3 DK3840753 T3 DK 3840753T3
- Authority
- DK
- Denmark
- Prior art keywords
- migalastat
- mutation
- disease
- methods
- patients carrying
- 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
- 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
Landscapes
- 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)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862719962P | 2018-08-20 | 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 |
|---|---|
| DK3840753T3 true DK3840753T3 (da) | 2024-10-28 |
Family
ID=65279726
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK19703514.0T DK3840753T3 (da) | 2018-08-20 | 2019-01-16 | Migalastat til anvendelse i fremgangsmåder til behandling af fabrys sygdom hos patienter, der har en mutation i gla-genet |
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)
| 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)
| 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 |
-
2019
- 2019-01-16 EP EP23202199.8A patent/EP4282472A3/en active Pending
- 2019-01-16 HU HUE19703514A patent/HUE068783T2/hu unknown
- 2019-01-16 HR HRP20241454TT patent/HRP20241454T1/hr unknown
- 2019-01-16 WO PCT/US2019/013761 patent/WO2020040806A1/en not_active Ceased
- 2019-01-16 EA EA202190569A patent/EA202190569A1/ru unknown
- 2019-01-16 IL IL325185A patent/IL325185A/en unknown
- 2019-01-16 EP EP23202183.2A patent/EP4289475A3/en active Pending
- 2019-01-16 EP EP19703514.0A patent/EP3840753B1/en active Active
- 2019-01-16 BR BR112021003137-5A patent/BR112021003137A2/pt unknown
- 2019-01-16 FI FIEP19703514.0T patent/FI3840753T3/fi active
- 2019-01-16 PT PT197035140T patent/PT3840753T/pt unknown
- 2019-01-16 EP EP23202185.7A patent/EP4299117A3/en active Pending
- 2019-01-16 SI SI201930852T patent/SI3840753T1/sl unknown
- 2019-01-16 PL PL19703514.0T patent/PL3840753T3/pl unknown
- 2019-01-16 CN CN201980069272.8A patent/CN112888439A/zh active Pending
- 2019-01-16 AU AU2019326252A patent/AU2019326252A1/en not_active Abandoned
- 2019-01-16 KR KR1020247003291A patent/KR20240017127A/ko not_active Ceased
- 2019-01-16 EP EP23202198.0A patent/EP4289476A3/en not_active Withdrawn
- 2019-01-16 KR KR1020247003292A patent/KR20240017128A/ko active Pending
- 2019-01-16 DK DK19703514.0T patent/DK3840753T3/da active
- 2019-01-16 ES ES19703514T patent/ES2993833T3/es active Active
- 2019-01-16 KR KR1020217008263A patent/KR20210046729A/ko active Pending
- 2019-01-16 CA CA3110065A patent/CA3110065A1/en active Pending
- 2019-01-16 JP JP2021509744A patent/JP2021535107A/ja active Pending
- 2019-01-16 RS RS20241181A patent/RS66094B1/sr unknown
- 2019-01-16 US US17/269,890 patent/US12594268B2/en active Active
- 2019-01-16 EP EP23202201.2A patent/EP4285991A3/en not_active Withdrawn
- 2019-01-16 SG SG11202101117QA patent/SG11202101117QA/en unknown
- 2019-01-16 SM SM20240436T patent/SMT202400436T1/it unknown
- 2019-01-16 MX MX2021002070A patent/MX2021002070A/es unknown
- 2019-01-16 LT LTEPPCT/US2019/013761T patent/LT3840753T/lt unknown
- 2019-01-16 EP EP23202200.4A patent/EP4285990A3/en active Pending
-
2021
- 2021-02-18 IL IL280961A patent/IL280961B1/en unknown
- 2021-02-19 CL CL2021000435A patent/CL2021000435A1/es unknown
- 2021-02-19 MX MX2024008350A patent/MX2024008350A/es unknown
-
2023
- 2023-04-25 JP JP2023071652A patent/JP7784398B2/ja active Active
- 2023-10-02 US US18/479,396 patent/US20240277687A1/en active Pending
-
2024
- 2024-03-13 CL CL2024000741A patent/CL2024000741A1/es unknown
- 2024-03-13 CL CL2024000739A patent/CL2024000739A1/es unknown
-
2025
- 2025-07-04 AU AU2025205141A patent/AU2025205141A1/en active Pending
- 2025-07-14 JP JP2025117816A patent/JP2025165955A/ja active Pending
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| DK3840753T3 (da) | Migalastat til anvendelse i fremgangsmåder til behandling af fabrys sygdom hos patienter, der har en mutation i gla-genet | |
| DK3984538T3 (da) | Migalastat til anvendelse til behandling af fabrys sygdom hos patienter med en mutation i gla-genet | |
| DK3364997T3 (da) | Aspartoacylase genterapi til behandling af canavans sygdom | |
| DK3673080T3 (da) | Antisense-oligomerer til behandling af lidelser og sygdomme | |
| IL282624A (en) | Methods and compositions for treatment of presbyopia, mydriasis, and other ocular disorders | |
| EP3562514A4 (en) | GENE THERAPY FOR THE TREATMENT OF WILSON'S DISEASE | |
| DK3858977T5 (da) | Stamme til forebyggelse og behandling af stofskiftesygdomme, og brugen heraf | |
| MA52709A (fr) | Administration d'adn | |
| IL283127A (en) | Animal models, screening methods, and treatment methods for intraocular diseases or disorders | |
| EP3829569C0 (en) | SPINOSYNE FORMULATIONS FOR USE IN THE TREATMENT OF EYE DISEASES CAUSED BY DEMODEX | |
| CY1120454T1 (el) | Παραγωγα 2,4-θειαζολιδινεδιονης στη θεραπεια διαταραχων του κεντρικου νευρικου συστηματος | |
| DK3526207T3 (da) | Heterocykliske forbindelser med aktivitet som modulatorer af muscarine m1- og/eller m4-receptorer i behandling af cns-sygdomme og smerter | |
| MA43388A (fr) | Mutagenèse ciblée d'acides nucléiques du type enzyme à pont berbérine du tabac | |
| SG11202011010YA (en) | 1,3-thiazol-2-yl substituted benzamides for the treatment of diseases associated with nerve fiber sensitization | |
| SG11202011018PA (en) | 1,3-thiazol-2-yl substituted benzamides for the treatment of diseases associated with nerve fiber sensitization | |
| MX2017009417A (es) | Formas solidas de acido 2-(5-(3-fluorofenil)-3-hidroxipicolinamido )acetico, composiciones y usos de las mismas. | |
| DK3749308T3 (da) | Behandling af patienter med klassisk fabrys sygdom med migalastat | |
| DK3419672T5 (da) | Genterapi til behandling af retinal degenerationssygdom | |
| EP4046609C0 (en) | EXTENDED PHARMACOLOGICAL RELEASE OPHTHALMIC FORMULATION AND ITS USES | |
| EA201992004A1 (ru) | Способы предотвращения или лечения офтальмологических заболеваний | |
| IL309393A (en) | Retinal disorders | |
| DK4114390T3 (da) | Fremgangsmåder til behandling af fabrys sygdom hos patienter med en mutation i gla-genet | |
| EP3372249C0 (en) | AAV/UPR-PLUS VIRUS, UPR-PLUS FUSION PROTEIN, GENETIC TREATMENT METHOD AND ITS USE IN THE TREATMENT OF NEURODEGENERATIVE DISEASES SUCH AS, AMONG OTHERS, PARKINSON'S DISEASE AND HUNTINGTON'S DISEASE | |
| DK3630114T3 (da) | Migalastat til behandling af patienter med fabrys sygdom, der har nedsat nyrefunktion | |
| DK3846788T3 (da) | Pde11-hæmmere til anvendelse i behandling af parkinsons sygdom |