At least 15 PDUFA dates on FDA’s calendar for June

FDA has set targets dates for decisions on at least 15 therapeutics applications in June, including a BLA for what could become the first gene therapy for hemophilia A to reach the U.S. market.

The decision on Roctavian valoctocogene roxaparvovec, an AAV5 gene therapy from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), is not due until the end of the month. FDA extended the target date in March after the company submitted three-year durability data from its Phase III trial, which the agency had requested last fall. At year three, patients’ mean annualized bleed rate was reduced 80% and factor VIII usage 94% from the baseline, indicating a high degree of durability at that point. If approved, it would become the first gene therapy available for hemophilia A in the U.S., though it would not be the drug’s first approval globally. The European Commission approved the Roctavian last August.  ...