Valoctocogene Roxaparvovec Offers Stable and Durable Bleed Control in Hemophilia A
BioMarin Pharmaceuticals released positive results from its global 3-year phase 3 study of valoctocogene roxaparvovec (Roctavian™) for patients with hemophilia A. With 134 participants, GENEr8-1 is the largest phase 3 study on gene therapy for hemophilia to date. “We continue to learn more about the durability, safety and efficacy of valoctocogene roxaparvovec,” said Steven Pipe,…