Drugs / Cipaglucosidase alfa
Cipaglucosidase alfa
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Pombiliti | — | 2023-03-20 | europa.eu ↗ |
Trials 6 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT02675465 | Apr 2016 → Aug 2024 | glycogen storage disease II | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 34 trials | ||||||
| Phase 3 | NCT04808505 | Jul 2023 → Jul 2027 expected | glycogen storage disease due to acid maltase deficiency, infantile onset | Amicus Therapeutics | Recruiting | No outcome recorded |
| Phase 3 | NCT03911505 | Feb 2020 → Jun 2026 overdue | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04138277 | Dec 2019 → Dec 2024 | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Completed | No outcome recorded |
| Phase 3 | NCT03729362 | Dec 2018 → Dec 2020 | glycogen storage disease due to acid maltase deficiency, late-onset | Amicus Therapeutics | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT03865836 | — | glycogen storage disease II | Amicus Therapeutics | Available | No outcome recorded |
Also used as a comparator or background therapy in 1 trial
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| — | NCT04327973 Background | — | glycogen storage disease due to acid maltase deficiency, infantile onset | Amicus Therapeutics | Available | No outcome recorded |
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 9 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Cipaglucosidase Alfa | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03911505 ↗ |
| Known as | AT GAA | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594329 ↗ |
| Known as | ATB200 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT04327973 ↗ |
| Known as | cipa | “Cipaglucosidase alfa (cipa) is an enzyme replacement therapy that aims to replace the missing enzyme.” PMID 41631150 ↗ Jan 2026 |
| Known as | Cipaglucosidase alfa-atga | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594329 ↗ |
| Known as | Pombiliti | ChEMBL registry synonym — accepted as the source's own label CHEMBL4594329 ↗ |
| Action | Restore | “Cipaglucosidase alfa (cipa) is an enzyme replacement therapy that aims to replace the missing enzyme.” PMID 41631150 ↗ Jan 2026 |
| Modality | Protein / enzyme biologic | “a highly targeted rhGAA (ATB200)” NCT02675465 ↗ |
| Route | Intravenous | “Sequential single ascending doses of intravenously infused ATB200” NCT02675465 ↗ |