HRP20250179T1 - Postupci liječenja fabrijeve bolesti kod pacijenta s mutacijom gla gena - Google Patents
Postupci liječenja fabrijeve bolesti kod pacijenta s mutacijom gla gena Download PDFInfo
- Publication number
- HRP20250179T1 HRP20250179T1 HRP20250179TT HRP20250179T HRP20250179T1 HR P20250179 T1 HRP20250179 T1 HR P20250179T1 HR P20250179T T HRP20250179T T HR P20250179TT HR P20250179 T HRP20250179 T HR P20250179T HR P20250179 T1 HRP20250179 T1 HR P20250179T1
- Authority
- HR
- Croatia
- Prior art keywords
- migalastat
- salt
- patient
- use according
- administered
- Prior art date
Links
- 238000000034 method Methods 0.000 title claims 3
- 206010064571 Gene mutation Diseases 0.000 title 1
- 101150014526 Gla gene Proteins 0.000 title 1
- 201000010099 disease Diseases 0.000 title 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 title 1
- 229950007469 migalastat Drugs 0.000 claims 17
- 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 17
- 150000003839 salts Chemical class 0.000 claims 16
- 239000012458 free base Substances 0.000 claims 3
- 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
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 1
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 1
- 102220419551 c.86C>A Human genes 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 229950010505 migalastat hydrochloride Drugs 0.000 claims 1
- 230000035772 mutation Effects 0.000 claims 1
- 102200001975 rs121908466 Human genes 0.000 claims 1
- 102220320590 rs1554306309 Human genes 0.000 claims 1
- 102220198259 rs749415085 Human genes 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
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
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)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Epidemiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Wood Science & Technology (AREA)
- Genetics & Genomics (AREA)
- Analytical Chemistry (AREA)
- Zoology (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Molecular Biology (AREA)
- Pathology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202062986297P | 2020-03-06 | 2020-03-06 | |
| EP21715035.8A EP4114390B1 (en) | 2020-03-06 | 2021-03-05 | Methods of treating fabry disease in patients having a mutation in the gla gene |
| PCT/US2021/020984 WO2021178737A1 (en) | 2020-03-06 | 2021-03-05 | Methods of treating fabry disease in patients having a mutation in the gla gene |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HRP20250179T1 true HRP20250179T1 (hr) | 2025-04-11 |
Family
ID=75267607
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HRP20250179TT HRP20250179T1 (hr) | 2020-03-06 | 2021-03-05 | Postupci liječenja fabrijeve bolesti kod pacijenta s mutacijom gla gena |
Country Status (25)
| Country | Link |
|---|---|
| US (1) | US20230136297A1 (https=) |
| EP (2) | EP4114390B1 (https=) |
| JP (2) | JP7749572B2 (https=) |
| KR (1) | KR20220152547A (https=) |
| CN (1) | CN115427040A (https=) |
| AR (1) | AR122390A1 (https=) |
| AU (1) | AU2021230371A1 (https=) |
| BR (1) | BR112022017881A2 (https=) |
| CA (1) | CA3170718A1 (https=) |
| CL (1) | CL2022002423A1 (https=) |
| DK (1) | DK4114390T3 (https=) |
| ES (1) | ES3013813T3 (https=) |
| FI (1) | FI4114390T3 (https=) |
| HR (1) | HRP20250179T1 (https=) |
| HU (1) | HUE070116T2 (https=) |
| IL (1) | IL296221A (https=) |
| LT (1) | LT4114390T (https=) |
| MX (1) | MX2022011030A (https=) |
| PL (1) | PL4114390T3 (https=) |
| PT (1) | PT4114390T (https=) |
| RS (1) | RS66530B1 (https=) |
| SI (1) | SI4114390T1 (https=) |
| SM (1) | SMT202500072T1 (https=) |
| TW (1) | TW202146020A (https=) |
| WO (1) | WO2021178737A1 (https=) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
Family Cites Families (3)
| 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 |
-
2021
- 2021-03-05 EP EP21715035.8A patent/EP4114390B1/en active Active
- 2021-03-05 AU AU2021230371A patent/AU2021230371A1/en active Pending
- 2021-03-05 EP EP24217104.9A patent/EP4527454A3/en active Pending
- 2021-03-05 JP JP2022553583A patent/JP7749572B2/ja active Active
- 2021-03-05 FI FIEP21715035.8T patent/FI4114390T3/fi active
- 2021-03-05 US US17/909,450 patent/US20230136297A1/en active Pending
- 2021-03-05 SI SI202130266T patent/SI4114390T1/sl unknown
- 2021-03-05 PT PT217150358T patent/PT4114390T/pt unknown
- 2021-03-05 AR ARP210100575A patent/AR122390A1/es unknown
- 2021-03-05 ES ES21715035T patent/ES3013813T3/es active Active
- 2021-03-05 DK DK21715035.8T patent/DK4114390T3/da active
- 2021-03-05 TW TW110107854A patent/TW202146020A/zh unknown
- 2021-03-05 CN CN202180028403.5A patent/CN115427040A/zh active Pending
- 2021-03-05 RS RS20250171A patent/RS66530B1/sr unknown
- 2021-03-05 BR BR112022017881A patent/BR112022017881A2/pt unknown
- 2021-03-05 HR HRP20250179TT patent/HRP20250179T1/hr unknown
- 2021-03-05 MX MX2022011030A patent/MX2022011030A/es unknown
- 2021-03-05 SM SM20250072T patent/SMT202500072T1/it unknown
- 2021-03-05 IL IL296221A patent/IL296221A/en unknown
- 2021-03-05 LT LTEPPCT/US2021/020984T patent/LT4114390T/lt unknown
- 2021-03-05 WO PCT/US2021/020984 patent/WO2021178737A1/en not_active Ceased
- 2021-03-05 CA CA3170718A patent/CA3170718A1/en active Pending
- 2021-03-05 PL PL21715035.8T patent/PL4114390T3/pl unknown
- 2021-03-05 KR KR1020227034499A patent/KR20220152547A/ko active Pending
- 2021-03-05 HU HUE21715035A patent/HUE070116T2/hu unknown
-
2022
- 2022-09-06 CL CL2022002423A patent/CL2022002423A1/es unknown
-
2025
- 2025-09-24 JP JP2025157586A patent/JP2026016375A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| TW202146020A (zh) | 2021-12-16 |
| RS66530B1 (sr) | 2025-03-31 |
| AR122390A1 (es) | 2022-09-07 |
| EP4114390A1 (en) | 2023-01-11 |
| EP4114390B1 (en) | 2024-12-04 |
| JP2023516753A (ja) | 2023-04-20 |
| LT4114390T (lt) | 2025-02-10 |
| US20230136297A1 (en) | 2023-05-04 |
| WO2021178737A1 (en) | 2021-09-10 |
| SMT202500072T1 (it) | 2025-03-12 |
| BR112022017881A2 (pt) | 2022-12-06 |
| MX2022011030A (es) | 2022-12-13 |
| JP7749572B2 (ja) | 2025-10-06 |
| AU2021230371A1 (en) | 2022-09-29 |
| KR20220152547A (ko) | 2022-11-16 |
| CA3170718A1 (en) | 2021-09-10 |
| FI4114390T3 (fi) | 2025-03-07 |
| IL296221A (en) | 2022-11-01 |
| CL2022002423A1 (es) | 2023-05-05 |
| PL4114390T3 (pl) | 2025-06-23 |
| PT4114390T (pt) | 2025-02-24 |
| CN115427040A (zh) | 2022-12-02 |
| EP4527454A2 (en) | 2025-03-26 |
| ES3013813T3 (en) | 2025-04-15 |
| JP2026016375A (ja) | 2026-02-03 |
| EP4527454A3 (en) | 2025-09-24 |
| SI4114390T1 (sl) | 2025-03-31 |
| HUE070116T2 (hu) | 2025-05-28 |
| DK4114390T3 (da) | 2025-02-24 |
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