Valoctocogene roxaparvovec - BioMarin Pharmaceutical

Drug Profile

Valoctocogene roxaparvovec - BioMarin Pharmaceutical

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

Latest Information Update: 02 Nov 2017

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At a glance

  • Originator St. Jude Childrens Research Hospital; University College London
  • Developer BioMarin Pharmaceutical
  • Class 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 I/II Haemophilia A

Most Recent Events

  • 26 Oct 2017 Valoctocogene roxaparvovec - BioMarin Pharmaceutical receives Breakthrough Therapy status for Haemophilia A in USA
  • 18 Oct 2017 Medicines and Healthcare Products Regulatory Agency (MHRA) approves IND application for valoctocogene roxaparvovec in Haemophilia A
  • 18 Oct 2017 US FDA approves IND application for valoctocogene roxaparvovec in Haemophilia A
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