FDA accepts BLA resubmission for valoctocogene roxaparvovec for severe hemophilia A
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The FDA accepted a biologics license application resubmission for valoctocogene roxaparvovec as treatment for adults with severe hemophilia A, according to a press release from the agent’s manufacturer.
The resubmitted BLA for valoctocogene roxaparvovec (BioMarin), an investigational adeno-associated virus (AAV) vector gene therapy, includes the company’s responses to all deficiencies listed in an FDA complete response letter issued 2 years ago, the press release stated.
"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, said in the press release. “In addition, we have proposed 15 years of follow-up for all clinical study participants, as well as a postapproval registry study to follow patients dosed in a real-world setting, to further characterize long-term effects on safety and efficacy that will contribute to increasing the body of knowledge of AAV gene therapy in severe hemophilia A.”
The BLA also includes 2-year outcomes of all participants in GENEr8-1, a global phase 3 study that showed durable and stable bleed control among people with severe hemophilia A, with reductions in mean annualized bleeding rate and mean annualized factor VIII infusion rate. Other supportive evidence comes from 5 years of follow-up of a cohort that received a dose of 613 vector genomes per kg in a phase 1/phase 2 dose-escalation study.
The single dose has been well tolerated to date, with no delayed-onset adverse events related to treatment, according to the press release. The most common adverse reactions include alanine aminotransferase elevation, aspartate aminotransferase elevation, nausea, headache and fatigue. No study participants developed factor VIII inhibitors, thromboembolic events or cancers associated with the gene therapy, the press release stated.
The FDA is expected to make a decision about approval of valoctocogene roxaparvovec for this indication by March 31, 2023. If approved, it would be the first gene therapy for severe hemophilia A in the United States, according to BioMarin.
Valoctocogene roxaparvovec has been granted FDA regenerative medicine advance therapy, breakthrough therapy and orphan drug designations.
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