Valoctocogene roxaparvovec - BioMarin Pharmaceutical

Drug Profile

Valoctocogene roxaparvovec - BioMarin Pharmaceutical

Alternative Names: AAV5-FVIII Gene Transfer - BioMarin; BMN-270

Latest Information Update: 24 May 2018

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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 Blood coagulation factor replacements; Factor X stimulants; 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

  • Phase III Haemophilia A

Most Recent Events

  • 15 May 2018 BioMarin plans a phase I/II Study for Haemophilia A with the 6 x 1013vg/vg dose in AAV5 positive patients in the first half of 2018 (NCT03520712)
  • 15 May 2018 BioMarin Pharmaceuticals initiates a phase I/II study (BMN 270-203) in Haemophilia A in United Kingdom (Parenteral) (NCT03520712)
  • 16 Apr 2018 Updated efficacy data from a phase I/II trial in Haemophilia A released by BioMarin
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