valoctocogene roxaparvovec
Trade name: Roctavian
Gene therapyapprovedOrphan Drug FDABreakthrough Therapy FDA
Approved
Jun 29, 2023
Clinical trial activity
7 trials · 1 clinical orgs · 1 marketing orgs
Phase 1
3
Phase 2
7
Phase 3
4
Phase 4
0
Earliest trial started Aug 1, 2015 (NCT02576795)
Timeline
Indications
Approved for
Mechanism of action
- Coagulation factor IXEXOGENOUS PROTEIN
Coagulation factor IX exogenous protein
AAV5 based vector containing the cDNA of the B-domain deleted SQ form of human coagulation factor VIII gene under the control of a liver-specific promoter
- Coagulation factor VIIISupplementation
F8 supplementation
Approval history
- approvedBreakthroughJun 29, 2023
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Availability
- Prescription Only
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL4297579 ↗
Also known as
- bmn 270
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06224907 | Phase 3 | Dec 25, 2023 | Biomarin |
| NCT04684940 | Phase 1/Phase 2 (Phase 2) | Dec 31, 2020 | Biomarin |
| NCT04323098 | Phase 3 | Jun 30, 2020 | Biomarin |
| NCT03520712 | Phase 1/Phase 2 (Phase 2) | Apr 3, 2018 | Biomarin |
| NCT03392974 | Phase 3 | Mar 14, 2018 | Biomarin |
| NCT03370913 | Phase 3 | Dec 19, 2017 | Biomarin |
| NCT02576795 | Phase 1/Phase 2 (Phase 2) | Aug 1, 2015 | Biomarin |
Showing 7 of 7 trials
Page 1 / 1
Organizations
Research & Development (1)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Biomarin | For profit | 7 | 7 | 2 | 2015 |
1 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| Biomarin | For profit | NDA | Jun 29, 2023 |