JPWO2023288210A5 - - Google Patents
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- Publication number
- JPWO2023288210A5 JPWO2023288210A5 JP2024501910A JP2024501910A JPWO2023288210A5 JP WO2023288210 A5 JPWO2023288210 A5 JP WO2023288210A5 JP 2024501910 A JP2024501910 A JP 2024501910A JP 2024501910 A JP2024501910 A JP 2024501910A JP WO2023288210 A5 JPWO2023288210 A5 JP WO2023288210A5
- Authority
- JP
- Japan
- Prior art keywords
- patient
- migalastat
- pharmaceutical composition
- administered
- day
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 238000000034 method Methods 0.000 claims 13
- 239000008194 pharmaceutical composition Substances 0.000 claims 7
- 229950007469 migalastat Drugs 0.000 claims 6
- 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 6
- 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 3
- 229950010505 migalastat hydrochloride Drugs 0.000 claims 3
- 230000000144 pharmacologic effect Effects 0.000 claims 3
- 150000003839 salts Chemical class 0.000 claims 3
- 208000024720 Fabry Disease Diseases 0.000 claims 2
- 102000005840 alpha-Galactosidase Human genes 0.000 claims 2
- 108010030291 alpha-Galactosidase Proteins 0.000 claims 2
- 239000003814 drug Substances 0.000 claims 2
- 238000002641 enzyme replacement therapy Methods 0.000 claims 2
- 238000002372 labelling Methods 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 239000006186 oral dosage form Substances 0.000 claims 2
- 108060006698 EGF receptor 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
- 238000009472 formulation Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 238000005303 weighing Methods 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163220816P | 2021-07-12 | 2021-07-12 | |
| US63/220,816 | 2021-07-12 | ||
| PCT/US2022/073626 WO2023288210A1 (en) | 2021-07-12 | 2022-07-12 | Methods of treating fabry disease in pediatric patients |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2024525760A JP2024525760A (ja) | 2024-07-12 |
| JP2024525760A5 JP2024525760A5 (https=) | 2025-04-28 |
| JPWO2023288210A5 true JPWO2023288210A5 (https=) | 2025-04-28 |
Family
ID=83149437
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024501910A Pending JP2024525760A (ja) | 2021-07-12 | 2022-07-12 | 小児患者のファブリー病を治療する方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20240293387A1 (https=) |
| EP (1) | EP4370120A1 (https=) |
| JP (1) | JP2024525760A (https=) |
| KR (1) | KR20240034203A (https=) |
| AU (1) | AU2022310691A1 (https=) |
| CA (1) | CA3225511A1 (https=) |
| WO (1) | WO2023288210A1 (https=) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
-
2022
- 2022-07-12 EP EP22761857.6A patent/EP4370120A1/en active Pending
- 2022-07-12 US US18/578,483 patent/US20240293387A1/en active Pending
- 2022-07-12 WO PCT/US2022/073626 patent/WO2023288210A1/en not_active Ceased
- 2022-07-12 AU AU2022310691A patent/AU2022310691A1/en active Pending
- 2022-07-12 KR KR1020247003776A patent/KR20240034203A/ko active Pending
- 2022-07-12 JP JP2024501910A patent/JP2024525760A/ja active Pending
- 2022-07-12 CA CA3225511A patent/CA3225511A1/en active Pending
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