cipaglucosidase alfa
Trade name: Pombiliti
Recombinant proteinapprovedOrphan Drug FDABreakthrough Therapy FDA
Approved
Sep 28, 2023
Clinical trial activity
1 trials · 1 clinical orgs · 1 marketing orgs
Phase 1
0
Phase 2
1
Phase 3
1
Phase 4
0
Earliest trial started Dec 31, 2021 (NCT04808505)
Timeline
2010s
- Sep 13, 2017
Orphan Drug Designation
2020s
- Jan 1, 2021
Amicus Therapeutics — Earliest Phase 3 Sponsor(trial)
- Sep 28, 2023
Amicus Therapeutics — Earliest FDA Approval
- Sep 28, 2023
Amicus Therapeutics — Marketing Organization
- Sep 28, 2023
Amicus Therapeutics — NDA Organization
Indications
Approved for
Studied for
Mechanism of action
- GlycogenHYDROLYTIC ENZYME
Glycogen hydrolytic enzyme
Provides an exogenous source of lysosomal acid alpha-glucosidase.
- Neutral alpha-glucosidase ABSupplementation
GANAB supplementation
Approval history
- approvedBreakthroughSep 28, 2023
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Parenteral
- Availability
- Prescription Only
Oral
No
Parenteral
Yes
Topical
No
Black box warning
Sources
- ChEMBLCHEMBL4594329 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04808505 | Phase 3 | Dec 31, 2021 | Amicus Therapeutics |
Showing 1 of 1 trials
Page 1 / 1
Organizations
Research & Development (1)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Amicus Therapeutics | For profit | 1 | 1 | 1 | 2021 |
1 organizations
Page 1 / 1
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Amicus Therapeutics | For profit | MKTG | Sep 28, 2023 |
| Amicus Therapeutics | For profit | NDA | Sep 28, 2023 |