FDA issues complete response letter for valoctocogene roxaparvovec gene therapy for severe hemophilia A. BioMarin.

BioMarin Pharmaceutical Inc. announced that the FDA issued a Complete Response Letter (CRL) to the Company's Biologics License Application (BLA) for valoctocogene roxaparvovec gene therapy for severe hemophilia A on August 18, 2020. The FDA issues a CRL to indicate that the review cycle for an application is complete and that the application is not ready for approval in its present form. Having previously agreed with the Agency on the extent of data necessary to support the BLA, the FDA introduced a new recommendation for two years of data from the Company's ongoing 270-301 study (Phase III) to provide substantial evidence of a durable effect using Annualized Bleeding Rate (ABR) as the primary endpoint. The Agency first informed the Company of this recommendation in the CRL having not raised this at any time during development or review. The Agency recommended that the Company complete the Phase III Study and submit two-year follow-up safety and efficacy data on all study participants. FDA concluded that the differences between Study 270-201 (Phase 1/II) and the Phase III study limited its ability to rely on the Phase 1/II study to support durability of effect. The Phase III study was fully enrolled in November 2019, and the last patient will complete two years of follow up in November 2021. The Company plans to meet with the Agency in the coming weeks to align on the next steps to obtain approval.