Priority Review for BioMarin Gene Therapy

FDA has granted priority review to a BioMarin BLA for its investigational AAV5 gene therapy (valoctocogene roxaparvovec) for adults with hemophilia A. This is the first time FDA has accepted a marketing application for gene therapy for any type of hemophilia. The PDUFA action date for the BLA is 8/21. BioMarin says it has been told that FDA plans to schedule an advisory committee meeting to discuss the product.

The BLA is based on a Phase 3 interim analysis of study participants treated with the investigational product manufactured by the to-be-commercialized process and three-year Phase 1/2 data. The company says it has constructed, commissioned, and validated one of the first gene therapy manufacturing facilities of its kind in the world.

FDA also has accepted the BioMarin PMA application for an AAV5 total antibody assay intended as a companion diagnostic test for valoctocogene roxaparvovec.