BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Con...

BioMarin Announces Oral Presentation of Positive One-Year Results from Phase 3 Pivotal Trial with Valoctocogene Roxaparvovec Gene Therapy in Adults with Severe Hemophilia A at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress

Largest Gene Therapy Study in Hemophilia A Demonstrates Superiority in Key Clinical Efficacy Endpoints Compared to Baseline Factor VIII Prophylactic Therapy

Significantly Reduced Mean Annualized Bleeding Rate (ABR) by 84% (p-value