BioMarin Pharmaceuticals announced that the US Food and Drug Administration (FDA) has accepted their resubmission of the Biologics License Application (BLA) for valoctocogene roxaparvovec, a potential adeno-associated virus (AAV) gene therapy being investigated for the treatment of severe hemophilia A.

The therapy, if approved, would be the first gene therapy in the US to treat patients with severe hemophilia A.

“In this application, we have provided a substantial body of evidence that supports the safe and effective use of valoctocogene roxaparvovec for the treatment of adults with severe hemophilia A,” Hank Fuchs, MD, president of worldwide research and development at BioMarin, wrote. “In addition, we have proposed 15 years of follow-up for all clinical study participants, as well as a post-approval registry study to follow patients dosed in a real-world setting.”

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The BLA resubmission provides 2-year outcome data from patients in the GENEr8-1 phase 3 trial, a global study showing stable and durable reductions in mean annualized bleeding rate and factor VIII infusion rate. The resubmission also included 5-year follow-up data from a cohort in the ongoing phase 1/2 dose escalation study, along with proposals for a postapproval registry and a 15-year follow-up study for all participants.

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The FDA granted Regenerative Medicine Advanced Therapy status to valoctocogene roxaparvovec in March 2021, and the European Commission granted conditional marketing authorization to the potential treatment in August 2022.

Hemophilia A is an X-linked inherited condition in which patients lack factor VIII, which helps the blood clot. Valoctocogene roxaparvovec is designed to transduce a modified human factor VIII encoding sequence, which leads to the sustained production of endogenous factor VIII in these patients. To date, a single 6e13 vg/kg dose of the investigational therapy has been shown to be well tolerated and without treatment-associated adverse events or factor VIII inhibitor development.


FDA accepts BioMarin’s Biologics License Application (BLA) for valoctocogene roxaparvovec AAV gene therapy for adults with severe hemophilia A. News release. BioMarin Pharmaceutical Inc; October 12, 2022.