CEMI - BioMarin's Hemophilia Data And Other News: The Good Bad And Ugly Of Biopharma
BioMarin Offers Additional Data for Valoctocogene Roxaparvovec Gene Therapy
BioMarin Pharmaceutical Inc. (BMRN) provided an addendum to its previously announced four year update of an investigational gene therapy for treating severe hemophilia A. The study involved six patients who were previously given Factor VIII prophylaxis in the 6e13 vg/kg cohort. The cutoff date for the data is April 8, 2020.
The data showed that six evaluable patients reported significant and sustained decline in bleeding that required Factor VIII infusions. The mean and median Annualized Bleed Rate ((ABR)) were reported at 16.3 and 16.5