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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

Latest Information Update: 04 Apr 2025

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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

  • Marketed Haemophilia A

Most Recent Events

  • 20 Nov 2024 BioMarin Pharmaceutical completes a phase III trial for Haemophilia A in Australia, Belgium, Brazil, England, France, Germany, Israel, Italy, Japan, Russia, Scotland, South Africa, South Korea, Spain, Taiwan, United Kingdom, USA (NCT03370913)
  • 07 Aug 2024 BioMarin Pharmaceuticals terminates a phase I/II study (BMN 270-203) in Haemophilia A in South Korea and Taiwan, France, United Kingdom and South Africa (IV) due to early rollover to long-term extension study (NCT03520712)
  • 07 Jun 2024 Efficacy and adverse events data from a phase III GENEr8-1 trial in Haemophilia A released by BioMarin Pharmaceutical

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