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Valoctocogene roxaparvovec - BioMarin Pharmaceutical

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Drug Profile

Valoctocogene roxaparvovec - BioMarin Pharmaceutical

Alternative Names: AAV5-hFVIII-SQ; BMN-270; Factor-VIII-gene-therapy-BioMarin; Roctavian; V-ROX

Latest Information Update: 26 Feb 2026

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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
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Haemophilia A
  • New Molecular Entity No

Highest Development Phases

  • Registered Haemophilia A

Most Recent Events

  • 24 Feb 2026 Withdrawn for Haemophilia A in Germany, Italy (IV)
  • 24 Jun 2025 Adverse events and efficacy data from the phase-III GENEr8-1 trial in Haemophilia A released by BioMarin Pharmaceutical
  • 08 May 2025 BioMarin Pharmaceutical completes the phase III GENEr8-3 trial in Haemophilia A in USA, Australia, Brazil and Taiwan (NCT04323098)

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