CA3242701A1 - Treatment of gm2 gangliosidosis - Google Patents
Treatment of gm2 gangliosidosisInfo
- Publication number
- CA3242701A1 CA3242701A1 CA3242701A CA3242701A CA3242701A1 CA 3242701 A1 CA3242701 A1 CA 3242701A1 CA 3242701 A CA3242701 A CA 3242701A CA 3242701 A CA3242701 A CA 3242701A CA 3242701 A1 CA3242701 A1 CA 3242701A1
- Authority
- CA
- Canada
- Prior art keywords
- gangliosidosis
- treatment
- compound
- formula
- specifically
- 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
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
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
- A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Life Sciences & Earth Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Neurology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Organic Chemistry (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Pyridine Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP22150783.3 | 2022-01-10 | ||
| EP22150783.3A EP4209215A1 (en) | 2022-01-10 | 2022-01-10 | Treatment of gm2 gangliosidosis |
| EP22193136 | 2022-08-31 | ||
| EP22193136.3 | 2022-08-31 | ||
| PCT/EP2023/050459 WO2023131720A1 (en) | 2022-01-10 | 2023-01-10 | Treatment of gm2 gangliosidosis |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3242701A1 true CA3242701A1 (en) | 2023-07-13 |
Family
ID=84982296
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3242701A Pending CA3242701A1 (en) | 2022-01-10 | 2023-01-10 | Treatment of gm2 gangliosidosis |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20250120958A1 (https=) |
| EP (2) | EP4714497A2 (https=) |
| JP (1) | JP2025501646A (https=) |
| AU (1) | AU2023205441A1 (https=) |
| CA (1) | CA3242701A1 (https=) |
| IL (1) | IL314212A (https=) |
| MX (1) | MX2024008583A (https=) |
| TW (1) | TW202337456A (https=) |
| WO (1) | WO2023131720A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| TW202539666A (zh) * | 2024-02-01 | 2025-10-16 | 荷蘭商雅捷法羅斯公司 | Gm1神經節苷脂儲積症之治療 |
| EP4595964A1 (en) * | 2024-02-01 | 2025-08-06 | Azafaros B.V. | Treatment of gm1 gangliosidosis |
| WO2026013261A1 (en) | 2024-07-11 | 2026-01-15 | Azafaros B.V. | Nizubaglustat for use in the treatment of lysosomal storage disorders |
| EP4678174A1 (en) | 2024-07-11 | 2026-01-14 | Azafaros B.V. | Nizubaglustat for use in the treatment of lysosomal storage disorders |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3461812B1 (en) | 2014-03-27 | 2021-10-13 | Academisch Medisch Centrum | N-(5-(biphen-4-ylmethyloxy)pentyl)-substituted iminosugars as inhibitors of glucosylceramide synthase |
| EP3978474A1 (en) * | 2020-10-02 | 2022-04-06 | Azafaros B.V. | Crystalline forms of a pharmaceutical compound |
-
2023
- 2023-01-10 EP EP25220465.6A patent/EP4714497A2/en active Pending
- 2023-01-10 MX MX2024008583A patent/MX2024008583A/es unknown
- 2023-01-10 JP JP2024539849A patent/JP2025501646A/ja active Pending
- 2023-01-10 EP EP23700690.3A patent/EP4463157B1/en active Active
- 2023-01-10 IL IL314212A patent/IL314212A/en unknown
- 2023-01-10 US US18/727,631 patent/US20250120958A1/en active Pending
- 2023-01-10 CA CA3242701A patent/CA3242701A1/en active Pending
- 2023-01-10 AU AU2023205441A patent/AU2023205441A1/en active Pending
- 2023-01-10 TW TW112101050A patent/TW202337456A/zh unknown
- 2023-01-10 WO PCT/EP2023/050459 patent/WO2023131720A1/en not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| WO2023131720A1 (en) | 2023-07-13 |
| TW202337456A (zh) | 2023-10-01 |
| EP4714497A2 (en) | 2026-03-25 |
| US20250120958A1 (en) | 2025-04-17 |
| JP2025501646A (ja) | 2025-01-22 |
| EP4463157A1 (en) | 2024-11-20 |
| IL314212A (en) | 2024-09-01 |
| MX2024008583A (es) | 2024-07-23 |
| EP4463157C0 (en) | 2026-02-25 |
| EP4463157B1 (en) | 2026-02-25 |
| AU2023205441A1 (en) | 2024-07-25 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W00-W00-W128 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: INACTIVE: IPC ASSIGNED Effective date: 20240702 Free format text: ST27 STATUS EVENT CODE: A-1-1-W00-W00-W129 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: INACTIVE: IPC REMOVED Effective date: 20240702 |
|
| P18 | Priority claim added or amended |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P18-P105 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIORITY CLAIM REQUIREMENTS DETERMINED COMPLIANT Effective date: 20240703 |
|
| W00 | Other event occurred |
Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT Effective date: 20240704 |
|
| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS Effective date: 20240725 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD Year of fee payment: 2 |
|
| U00 | Fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED Effective date: 20241206 |
|
| U11 | Full renewal or maintenance fee paid |
Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT Effective date: 20241206 Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL Effective date: 20241206 |