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HFA Responds to News of FDA Action on BioMarin Gene Therapy Application

Click Below to Translate Page to Spanish. [prisna-google-website-translator]Today BioMarin announced that the U.S. Food and Drug Administration has issued a letter indicating that BioMarin’s investigational gene therapy for severe hemophilia A is not ready for approval in its present form. BioMarin says the FDA has asked for two years of follow-up data showing evidence of […]

BioMarin Receives Complete Response Letter (CRL) from FDA for Gene Therapy for Severe Hemophilia A

Click Below to Translate Page to Spanish. [prisna-google-website-translator]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 […]

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