FDA Accepts Resubmission of BioMarin’s Gene Therapy Two Years After Rejection

The United States Food and Drug Administration accepted BioMarin’s resubmitted Biologics License Application (BLA) for valoctocogene roxaparvovec. The FDA rejected the gene therapy for adults with severe hemophilia A in 2020, citing a lack of data. Should the FDA approve the new submission, valoctocogene roxaparvovec would be the world’s first gene therapy treatment for hemophilia A.

Related Article: FDA Rejects BioMarin Pharmaceuticals’ Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

FDA Acceptance of a Prior Rejection

BioMarin initially submitted a BLA for valoctocogene roxaparvovec back in 2020. At the time, the company formed the basis of the submission on data from a Phase ½ study. Overall, participants in the study saw more than a 90% reduction in bleeding episodes.

However, in August of that year, the FDA issued a Complete Response Letter (CRL) to BioMarin’s BLA. Despite the agency and company having agreed prior on the extent of data required for the support of the BLA, the FDA added an addendum. The FDA requested that BioMarin complete their, at the time, ongoing Phase 3 trial alongside two-year follow-up safety and efficacy data. As such, the FDA branded with BLA with a rejection at the time. 

Doing just that, BioMarin did just that, resubmitting their BLA in September of this year. The requested Phase 3 data showed “stable and durable bleed control” alongside a lessened mean annualized bleeding rate. As a result, the FDA finally accepted the BLA.

On the acceptance, Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin, said, “This BLA resubmission is an important step that brings us closer to delivering a gene therapy treatment choice to address the unmet needs of people with severe hemophilia A in the United States. 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.”

Under the Prescription Drug User Fee Act (PDUFA), the target action date is set for March 31, 2023. Hopefully, after all the hoops BioMarin jumped through getting to this point, the FDA grants their treatment approval on that date. If they do, valoctocogene roxaparvovec would be the world’s first gene therapy treatment for hemophilia A.

Author

Max Heirich