BLA Submitted for Gene Therapy to Treat Hemophilia A

BioMarin has submitted a Biologics License Application (BLA) to the Food and Drug Administration (FDA) for valoctocogene roxaparvovec (BMN 270) for the treatment of hemophilia A in adults. This is the first marketing application submission for a gene therapy product for any type of hemophilia.

Valoctocogene roxaparvovec is an investigational adeno-associated virus (AAV) gene therapy that is administered as a single infusion to produce clotting factor VIII. The BLA submission is supported by interim analysis of a phase 3 study and 3-year phase 1/2 data. Results from the ongoing phase 1/2 study showed that bleed rate control and reduction in factor VIII usage was maintained for a third year following a single administration of valoctocogene roxaparvovec. 

The FDA previously granted Breakthrough Therapy and Orphan Drug designations to valoctocogene roxaparvovec. The Company anticipates the BLA review to commence in February 2020.

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“We look forward to working with the FDA as we seek marketing authorization for the potential first gene therapy for hemophilia A,” said Hank Fuchs, MD, President, Global Research and Development at BioMarin. “Our hope is one day very soon to deliver a transformative treatment that has the potential to change the way hemophilia A is treated.”

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