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# BioMarin Resubmitting Hemophilia A BLA in September
- URL: https://www.fdaweb.com/biomarin-resubmitting-hemophilia-a-bla-in-september/
- Published: 2022-05-31T12:00:00.000Z
- Updated: 2026-09-14T17:46:52.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5151936

BioMarin Pharmaceutical says it plans to resubmit a BLA for valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, by the end of September that will include previously reported results from the two-year follow-up safety and efficacy data from the Phase 3 GENEr8-1 study. In 8/2020, [FDA issued ](https://www.fdaweb.com/fda-rebuffs-biomarin-bla-for-hemophilia-gene-therapy/)a complete response letter for the BLA that requested two years of data from the Phase 3 study to provide evidence of a durable effect using annualized bleeding rate as the primary endpoint.

“Based on recent feedback received from the FDA related to BioMarin’s plans for the upcoming BLA, the agency has requested additional information and analyses of data to be included in the BLA prior to submission,” the company says. While the agency has not requested additional pre-clinical or clinical studies, according to the company, data on three years after dosing will become available during the expected BLA review. 

Additionally, Biomarin says a cohort dosed six years ago showed a 95% reduction in mean annualized bleed rate and 96% reduction in mean annualized Factor VIII Usage from baseline. And a five-year dosed cohort demonstrated a 91% reduction in mean annualized bleed rate and 93% reduction in mean annualized Factor VIII usage from baseline.