CA3252291A1 - METHODS OF TREATMENT FOR POMPE DISEASE - Google Patents
METHODS OF TREATMENT FOR POMPE DISEASEInfo
- Publication number
- CA3252291A1 CA3252291A1 CA3252291A CA3252291A CA3252291A1 CA 3252291 A1 CA3252291 A1 CA 3252291A1 CA 3252291 A CA3252291 A CA 3252291A CA 3252291 A CA3252291 A CA 3252291A CA 3252291 A1 CA3252291 A1 CA 3252291A1
- Authority
- CA
- Canada
- Prior art keywords
- rhgaa
- subject
- atb200
- mol
- miglustat
- 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
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/12—Carboxylic acids; Salts or anhydrides thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
- A61K47/38—Cellulose; Derivatives thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/0102—Alpha-glucosidase (3.2.1.20)
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)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Genetics & Genomics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Obesity (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Inorganic Chemistry (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Dermatology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (19)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263338601P | 2022-05-05 | 2022-05-05 | |
| US63/338,601 | 2022-05-05 | ||
| US202263399498P | 2022-08-19 | 2022-08-19 | |
| US202263399491P | 2022-08-19 | 2022-08-19 | |
| US63/399,498 | 2022-08-19 | ||
| US63/399,491 | 2022-08-19 | ||
| US202263404475P | 2022-09-07 | 2022-09-07 | |
| US63/404,475 | 2022-09-07 | ||
| US202263414897P | 2022-10-10 | 2022-10-10 | |
| US63/414,897 | 2022-10-10 | ||
| US202263428970P | 2022-11-30 | 2022-11-30 | |
| US63/428,970 | 2022-11-30 | ||
| US202263431920P | 2022-12-12 | 2022-12-12 | |
| US63/431,920 | 2022-12-12 | ||
| US202263434791P | 2022-12-22 | 2022-12-22 | |
| US63/434,791 | 2022-12-22 | ||
| US202363447222P | 2023-02-21 | 2023-02-21 | |
| US63/447,222 | 2023-02-21 | ||
| PCT/US2023/066657 WO2023215865A1 (en) | 2022-05-05 | 2023-05-05 | Methods for treating pompe disease |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3252291A1 true CA3252291A1 (en) | 2023-11-09 |
Family
ID=86692686
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA3252291A Pending CA3252291A1 (en) | 2022-05-05 | 2023-05-05 | METHODS OF TREATMENT FOR POMPE DISEASE |
Country Status (13)
| Country | Link |
|---|---|
| US (1) | US20260041744A1 (enExample) |
| EP (1) | EP4518885A1 (enExample) |
| JP (1) | JP2025515159A (enExample) |
| KR (1) | KR20250007602A (enExample) |
| CN (1) | CN119343147A (enExample) |
| AU (1) | AU2023265155A1 (enExample) |
| CA (1) | CA3252291A1 (enExample) |
| CL (1) | CL2024003377A1 (enExample) |
| CO (1) | CO2024016724A2 (enExample) |
| IL (1) | IL316776A (enExample) |
| MX (1) | MX2024013673A (enExample) |
| TW (1) | TW202400212A (enExample) |
| WO (1) | WO2023215865A1 (enExample) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2026006625A1 (en) | 2024-06-26 | 2026-01-02 | Amicus Therapeutics, Inc. | Miglustat formulations comprising nitrosamine for use in the treatment of pompe disease |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6534300B1 (en) | 1999-09-14 | 2003-03-18 | Genzyme Glycobiology Research Institute, Inc. | Methods for producing highly phosphorylated lysosomal hydrolases |
| WO2009102895A2 (en) | 2008-02-12 | 2009-08-20 | Amicus Therapeutics, Inc. | Method to predict response to pharmacological chaperone treatment of diseases |
| US11382980B2 (en) | 2013-10-08 | 2022-07-12 | Taiyo Kagaku Co., Ltd. | Oil/fat composition containing polyunsaturated fatty acid |
| JP6851964B2 (ja) * | 2014-09-30 | 2021-03-31 | アミカス セラピューティックス インコーポレイテッド | 炭水化物が増大している非常に強力な酸性アルファ−グルコシダーゼ |
| IL299470B2 (en) * | 2015-12-30 | 2026-05-01 | Amicus Therapeutics Inc | Improved acid alpha-glucosidase for the treatment of Pompe disease |
| CA3019354A1 (en) | 2016-03-30 | 2017-10-05 | Amicus Therapeutics, Inc. | Method for selection of high m6p recombinant proteins |
| KR102618519B1 (ko) * | 2016-03-30 | 2023-12-28 | 아미쿠스 세라퓨틱스, 인코포레이티드 | 재조합 산 알파-글루코시다제를 포함하는 제형 |
| CN119113091A (zh) * | 2017-05-15 | 2024-12-13 | 阿米库斯治疗学公司 | 重组人类酸性α-葡萄糖苷酶 |
| WO2020163480A1 (en) | 2019-02-05 | 2020-08-13 | Amicus Therapeutics, Inc. | Recombinant human acid alpha-glucosidase and uses thereof |
| JP2024506346A (ja) * | 2021-02-11 | 2024-02-13 | アミカス セラピューティックス インコーポレイテッド | 組換えヒト酸性アルファグルコシダーゼ及びその使用 |
-
2023
- 2023-05-05 WO PCT/US2023/066657 patent/WO2023215865A1/en not_active Ceased
- 2023-05-05 KR KR1020247039256A patent/KR20250007602A/ko active Pending
- 2023-05-05 AU AU2023265155A patent/AU2023265155A1/en active Pending
- 2023-05-05 CA CA3252291A patent/CA3252291A1/en active Pending
- 2023-05-05 EP EP23728984.8A patent/EP4518885A1/en active Pending
- 2023-05-05 TW TW112116800A patent/TW202400212A/zh unknown
- 2023-05-05 JP JP2024565157A patent/JP2025515159A/ja active Pending
- 2023-05-05 CN CN202380045183.6A patent/CN119343147A/zh active Pending
- 2023-05-05 IL IL316776A patent/IL316776A/en unknown
- 2023-05-05 US US18/863,111 patent/US20260041744A1/en active Pending
-
2024
- 2024-11-05 MX MX2024013673A patent/MX2024013673A/es unknown
- 2024-11-05 CL CL2024003377A patent/CL2024003377A1/es unknown
- 2024-12-05 CO CONC2024/0016724A patent/CO2024016724A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| MX2024013673A (es) | 2024-12-06 |
| IL316776A (en) | 2025-01-01 |
| CN119343147A (zh) | 2025-01-21 |
| AU2023265155A1 (en) | 2024-12-12 |
| KR20250007602A (ko) | 2025-01-14 |
| WO2023215865A1 (en) | 2023-11-09 |
| CL2024003377A1 (es) | 2025-03-28 |
| US20260041744A1 (en) | 2026-02-12 |
| CO2024016724A2 (es) | 2025-04-28 |
| JP2025515159A (ja) | 2025-05-13 |
| EP4518885A1 (en) | 2025-03-12 |
| TW202400212A (zh) | 2024-01-01 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20250057925A1 (en) | Recombinant Human Acid Alpha-Glucosidase | |
| WO2020163480A1 (en) | Recombinant human acid alpha-glucosidase and uses thereof | |
| US20240197839A1 (en) | Recombinant Human Acid Alpha-Glucosidase and Uses Thereof | |
| US20260041744A1 (en) | Methods for Treating Pompe Disease | |
| AU2023406479A1 (en) | Methods for treating late onset pompe disease in pediatric patients | |
| AU2023406510A1 (en) | Fexamethods for treating infantile-onset pompe disease in pediatric patients | |
| CN117157095A (zh) | 重组人类酸性α-葡萄糖苷酶和其用途 | |
| HK40026823B (en) | Recombinant human acid alpha-glucosidase | |
| HK40026823A (en) | Recombinant human acid alpha-glucosidase | |
| EA045409B1 (ru) | Рекомбинантная человеческая кислая альфа-глюкозидаза |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A00 | Application filed |
Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A00-A101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION RECEIVED - PCT Effective date: 20241105 |
|
| A15 | Pct application entered into the national or regional phase |
Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A15-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: NATIONAL ENTRY REQUIREMENTS DETERMINED COMPLIANT Effective date: 20250204 |
|
| 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: 20250204 |
|
| A00 | Application filed |
Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A00-A102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: COMPLIANCE REQUIREMENTS DETERMINED MET Effective date: 20250206 |
|
| 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: 20250206 |
|
| 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: 20250217 |
|
| 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: 20250407 |
|
| 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 PAID IN FULL Effective date: 20250407 |
|
| MFA | Maintenance fee for application paid |
Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD Year of fee payment: 3 |
|
| 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: 20260401 |
|
| 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 PAID IN FULL Effective date: 20260401 |