BioMarin gene therapy shows sustained benefit in hemophilia A study

BioMarin Pharmaceutical (NASDAQ:BMRN) announces updated data from an open-label Phase 1/2 clinical trial evaluating a single administration of gene therapy valoctocogene roxaparvovec in adults with severe hemophilia A. The results will be virtually presented at the World Federation of Hemophilia Summit June 14-19.

The mean annualized bleed rate (ABR) at year 4 in the 6e13 vg/kg cohort was 1.3. Six of seven participants experienced no spontaneous bleeds over the past year.

Mean ABR at year 3 in the 4e13 vg/kg cohort was 0.5. Five of six participants experienced no spontaneous bleeds over the past year.

Factor VIII activity levels remain in a range for efficacy in stopping/preventing bleeding.

No new safety signals have been reported and no patient has developed inhibitors to Factor VIII, experienced blood clotting events or withdrawn from the study.

The company’s U.S. marketing application is currently under FDA review with an action date of August 21 under Breakthrough Therapy and Orphan Drug status. Its application in Europe is under EMA review under accelerated assessment and Orphan Drug status.

https://seekingalpha.com/news/3578852-biomarin-gene-therapy-shows-sustained-benefit-in-hemophilia-study