BioMarin

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 | UCLB News

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, 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 haemophilia A. This is the largest and longest global Phase 3 study to date for any gene therapy in haemophilia with 134 participants.

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