BioMarin announces stable and durable annualised bleed control for ROCTAVIAN™ in largest Phase 3 gene therapy study in adults with severe haemophilia A

9 January 2023


BioMarin, a global biotechnology company dedicated to transforming lives through genetic discovery, has 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 haemophilia A. With 134 participants, this is the largest and longest global Phase 3 study to date for any gene therapy in haemophilia.

Read more from BioMarin here.