BioMarin Provides Positive Clinical Data for Hemophilia Therapy Trial
BioMarin Pharmaceutical (BMRN) reported data from its Phase 1/2 clinical trial for valoctocogene roxaparvovec in adults suffering from severe hemophilia A. The company reported that its US marketing application for the drug candidate is currently being reviewed by the FDA, and the decision is likely to be out by August 21, 2020. The drug candidate has been conferred Orphan Drug and Breakthrough Therapy status. The application is being treated under Priority Review pathway.
Valoctocogene roxaparvovec is an investigational gene therapy. The 4-year update has been presented