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BioMarin Announced Stable and Durable Annualized Bleed Control for ROCTAVIAN in Largest Phase 3 Gene Therapy Study in Adults with Severe Hemophilia A; Said 92% of Patients off Prophylaxis at the End of Year 3

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced positive results from more than three years of follow up from its ongoing global Phase 3 GENEr8-1 study of ROCTAVIANTM (valoctocogene roxaparvovec), an investigational one-time gene therapy for the treatment of adults with severe hemophilia A.

BMRN