Drug Profile
Valoctocogene roxaparvovec - BioMarin Pharmaceutical
Alternative Names: AAV5-hFVIII-SQ; BMN-270; Factor-VIII-gene-therapy-BioMarin; RoctavianLatest Information Update: 01 Feb 2024
Price :
$50
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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
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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
Most Recent Events
- 25 Dec 2023 Phase-III clinical trials in Haemophilia A (Treatment-experienced) in Japan (IV) (NCT06224907)
- 09 Dec 2023 Efficacy data from a phase III trial in Haemophilia A presented at the 65th American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2023)
- 31 Jul 2023 BioMarin plans to launch valoctocogene roxaparvovec for Haemophilia A in Italy and France