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A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

Trial Profile

A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions

Status: Active, no longer recruiting
Phase of Trial: Phase III

Latest Information Update: 05 Dec 2019

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At a glance

  • Drugs Valoctocogene-roxaparvovec (Primary)
  • Indications Haemophilia A
  • Focus Registrational; Therapeutic Use
  • Sponsors BioMarin Pharmaceutical
  • Most Recent Events

    • 03 Dec 2019 Status changed from recruiting to active, no longer recruiting.
    • 21 Nov 2019 According to a BioMarin Pharmaceutical media release, the company is on track to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of the year 2019.
    • 21 Nov 2019 According to a BioMarin Pharmaceutical media release, the company has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for valoctocogene roxaparvovec, for adults with severe hemophilia A. Subject to completion of EMA's validation check, the Company anticipates the start of the MAA review to commence in January 2020 under accelerated assessment. BioMarin will provide an update in January 2020.
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