BioMarin Receives European Orphan Drug Designation for BMN 270, First Investigational AAV-Factor VIII Gene Therapy for Patients with Hemophilia A

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced that BMN 270, an investigational gene therapy for the treatment of hemophilia A, has been granted orphan drug designation by the European Commission. In the European Union, orphan drug designation is given to treatments that are intended for life-threatening or chronically-debilitating conditions with a prevalence of not more than 5 in 10,000 people. Earlier this month, BioMarin announced BMN 270 had also received orphan drug designation from the U.S. Food and Drug Administration.
According to the news:

The Phase 1/2 study will evaluate the safety and efficacy of BMN 270 gene therapy in up to 12 patients with severe hemophilia A. The primary endpoints are to assess the safety of a single intravenous administration of a recombinant AAV, human-coagulation factor VIII vector and to determine the change from baseline of factor VIII expression level at 16 weeks after infusion. The kinetics, duration and magnitude of AAV-mediated factor VIII activity in individuals with hemophilia A will be determined and correlated to an appropriate BMN 270 dose.

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