The Food and Drug Administration has accepted for Priority Review the Biologics License Application (BLA) for Biomarin’s investigational adeno-associated virus (AAV) gene therapy, valoctocogene roxaparvovec, for the treatment of hemophilia A in adults. The Agency also accepted the premarket approval (PMA) application for an AAV5 total antibody assay, a companion diagnostic test for valoctocogene roxaparvovec.

The acceptance of the marketing application is the first for a gene therapy product for any type of hemophilia in the US. The Agency has set a Prescription Drug User Fee Act (PDUFA) action date of August 21, 2020. 

The application is based on a phase 3 interim analysis 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 acceptance of the filing and initiation of review for the first gene therapy for hemophilia A builds on years of scientific achievements in improving the standard of care for people with bleeding disorders,” said Doris V. Quon MD, Medical Director, Orthopaedic Hemophilia Treatment Center at The Orthopaedic Institute for Children.

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Priority Review designation is granted to drugs that, if approved, would provide significant improvements in safety or effectiveness for the treatment, diagnosis, or prevention of serious conditions. 

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