Valoctocogene roxaparvovec - BioMarin Pharmaceutical
Alternative Names: AAV5-hFVIII-SQ; BMN-270; Factor-VIII-gene-therapy-BioMarin; RoctavianLatest Information Update: 14 Jun 2024
At a glance
- Originator St. Jude Childrens Research Hospital; University College London
- Developer BioMarin Pharmaceutical
- Class Antihaemorrhagics; Blood coagulation factors; Gene therapies
- Mechanism of Action Factor VIII replacements; Gene transference
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Marketed Haemophilia A