SAN RAFAEL, Calif., Aug. 19, 2020 /PRNewswire/ — BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (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 3) 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

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