BioMarin Resubmits BLA for Hemophilia A Drug Candidate
BioMarin has resubmitted its Biologics License Application (BLA) for adeno-associated virus (AAV) gene therapy valoctocogene roxaparvovec in adult patients with severe hemophilia A.
The resubmission followed a Complete Response Letter the company received from the FDA in August 2020, declining to approval the BLA because of insufficient data. The agency recommended two years of data from a phase 3 study to provide substantial evidence of a durable effect using the Annualized Bleeding Rate as the primary endpoint.
The resubmission includes two-year outcomes from a global phase 3 study and supportive data from five years of follow-up from an ongoing dose escalation study, the company said.
BioMarin expects a response from the FDA by the end of October on whether the agency will accept the BLA resubmission for review.