Eladocagene Exuparvovec
Trade name: Kebilidi
Gene therapyapprovedOrphan Drug FDA
Approved
Nov 13, 2024
Clinical trial activity
2 trials · 2 clinical orgs · 1 marketing orgs
Phase 1
1
Phase 2
2
Phase 3
0
Phase 4
0
Earliest trial started Oct 1, 2014 (NCT01395641)
Timeline
2010s
- Jan 1, 2014
Earliest Phase 1 Sponsor(trial)
- Jun 8, 2016
Orphan Drug Designation
2020s
- Jan 1, 2021
PTC Therapeutics — Earliest Phase 2 Sponsor(trial)
- Nov 13, 2024
PTC Therapeutics — Earliest FDA Approval
- Nov 13, 2024
PTC Therapeutics — NDA Organization
Indications
Mechanism of action
- DDCEXOGENOUS GENE
DDC exogenous gene
AAV2 based vector containing the cDNA of the human dopa decarboxylase (DDC) gene under the control of the cytomegalovirus immediate-early promoter
Approval history
- approvedAcceleratedNov 13, 2024
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Availability
- Prescription Only
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL4298189 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT04903288 | Phase 2 | May 12, 2021 | PTC Therapeutics |
| NCT01395641 | Phase 1/Phase 2 (Phase 2) | Oct 1, 2014 | National Taiwan University |
Showing 2 of 2 trials
Page 1 / 1
Organizations
Research & Development (2)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| National Taiwan University | Academic/Hospital | 1 | 1 | 1 | 2014 |
| PTC Therapeutics | For profit | 1 | 1 | 1 | 2021 |
2 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| PTC Therapeutics | For profit | NDA | Nov 13, 2024 |