BMN 270

Drug Profile

BMN 270

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

Latest Information Update: 10 Sep 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

  • 02 Aug 2017 Updated efficacy data from a phase I/II trial in Haemophilia A released by BioMarin Pharmaceutical
  • 02 Aug 2017 Updated efficacy and adverse event data from a phase I/II trial in Haemophilia A released by BioMarin Pharmaceutical
  • 02 Aug 2017 BioMarin plans a phase III trial (4 x 1013 vg/kg dose) for Haemophilia A in USA
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