FDA Accepts BioMarin’s BLA Resubmission of AAV Gene Therapy for Severe Hemophilia A

The FDA has accepted BioMarin Pharmaceutical’s resubmission of a Biologics License Application (BLA) for its  investigational adeno-associated virus (AAV) gene therapy valoctocogene roxaparvovec for adult patients with severe hemophilia A, which includes frequent spontaneous hemorrhage and abnormal bleeding.

The company had previously received a Complete Response Letter (CRL) in August 2020, rejecting the previous BLA. The FDA recommended that the company provide two years of data for its ongoing phase 3 study as evidence of a durable effect using annualized bleeding rate as the primary endpoint.

The BLA resubmission includes a proposed long-term extension study following all clinical trial participants for up to 15 years, as well as a post-approval registry study to follow patients dosed in a real-world setting, the company said.

If approved, valoctocogene roxaparvovec would be the first gene therapy approved in the U.S. for the treatment of severe hemophilia A. The FDA’s target action date for the resubmitted application is March 31, 2023.