BioMarin Filing Additional Data for Hemophilia Gene Therapy

BioMarin Pharmaceutical is submitting to FDA additional long-term data (three years) as part of the ongoing BLA review for Roctavian (valoctocogene roxaparvovec), an investigational one-time gene therapy for treating adults with severe hemophilia A. The data submitted show the mean annualized bleed rate reduced by 80% from baseline and Factor VIII usage reduced by 94% in year three compared to baseline, according to the company. Additionally, it says 92% of treated patients remained off their prophylaxis treatments at the end of year three.

BioMarin says FDA has recently completed a pre-license inspection of the manufacturing facility, and it has provided responses to the comments and observations received at the close of the inspection, which it adds “all are addressable.” The user fee review action target date is 3/31, but is could be extended after the new data are submitted.