BioMarin Late-stage Trial On Valoctocogene Roxaparvovec Shows Superiority To Prophylaxis

BioMarin Pharmaceutical Inc. (BMRN) Monday announced new data for valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, in its positive pivotal study, GENEr8-1, during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.

The pivotal study demonstrated superiority to Factor VIII prophylaxis in key clinical efficacy endpoints. With 134 participants, this is the largest global Phase 3 study to date for gene therapy in hemophilia. All participants in the study received a single dose of valoctocogene roxaparvovec and completed a year or more of follow-up.

New data presented at ISTH include more details on annualized bleeding rate (ABR) in all study participants and annualized Factor VIII utilization rate, in terms of international units per kilogram per year of replacement Factor VIII. Over 90 percent of all participants in the GENEr8-1 study had an annualized bleed rate of zero or a lower bleed rate than baseline after week 4 after treatment with valoctocogene roxaparvovec.

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