> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# BioMarin Refiling BLA for Hemophilia A Gene Therapy
- URL: https://www.fdaweb.com/biomarin-refiling-bla-for-hemophilia-a-gene-therapy/
- Published: 2022-08-25T12:00:00.000Z
- Updated: 2026-09-14T17:57:16.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5152596

BioMarin Pharmaceutical says it will resubmit by the end of September its BLA for hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec) gene therapy after announcing the European Commission just granted conditional marketing authorization for the treatment. “Typically, BLA resubmissions are followed by a six-month review procedure,” BioMarin says. “However, the company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.”

BioMarin notes that the resubmission 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 said in May. 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.