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# FDA Rebuffs BioMarin BLA for Hemophilia Gene Therapy
- URL: https://www.fdaweb.com/fda-rebuffs-biomarin-bla-for-hemophilia-gene-therapy/
- Published: 2020-08-19T12:00:00.000Z
- Updated: 2026-09-14T16:25:30.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5147467

FDA has issued an 8/18 complete response letter to BioMarin Pharmaceutical for its valoctocogene roxaparvovec gene therapy for severe hemophilia A. The company [says](https://www.prnewswire.com/news-releases/biomarin-receives-complete-response-letter-crl-from-fda-for-valoctocogene-roxaparvovec-gene-therapy-for-severe-hemophilia-a-301114587.html?ref=fdaweb.com) the agency is now requesting two years of data from the ongoing Phase 3 study to provide evidence of a durable effect using annualized bleeding rate as the primary endpoint. BioMarin says the last patient enrolled in the study will complete two years of follow-up in 11/2021.

BioMarin says the valoctocogene roxaparvovec trials will continue while the company meets with FDA officials to discuss the next steps toward approval.