BioMarin Unveils News Data From Late-Stage Hemophilia A Gene Therapy Candidate

BioMarin Pharmaceutical Inc BMRN has announced new data from GENEr8-1 Phase 3 study for valoctocogene roxaparvovec, investigational gene therapy for severe hemophilia A.
Data were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress.
Top-line one-year results from this study were previously communicated in January 2021.
New data presented include more details on annualized bleeding rate (ABR) in all study participants and annualized Factor VIII utilization rate.
Over 90% (N=134) of all participants in the GENEr8-1 study had an ABR of zero or a lower bleed rate than baseline after week four after the treatment.
Mean annualized Factor VIII utilization rate decreased from baseline on Factor VIII prophylaxis by 99% from 3961.2 to 56.9 after week four after treatment with valoctocogene roxaparvovec.
As previously shared in January 2021, a single dose of valoctocogene roxaparvovec significantly reduced mean ABR by 84% from a prospectively collected 4.8 baseline to 0.8 bleeding episodes per year.
Study participants also experienced a clinically meaningful increase in endogenous Factor VIII expression.
Last week, the European Medicines Agency validated BioMarin's resubmission of a marketing application for the gene therapy.