BioMarin plans regulatory submissions for valoctocogene roxaparvovec to treat severe hemophilia A in 4Q 2019 in both U.S. and Europe

BioMarin Pharmaceutical Inc.announced that based on recent meetings with health authorities in the U.S. and Europe, the company plans to submit marketing applications to both the FDA and the European Medicines Agency (EMA) in 4Q 2019 for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A.

These submissions will be based on the updated three-year Phase 1/II data and the recently completed Phase III interim analysis of patients treated with material from the to-be-commercialized process. Both submissions are expected to represent the first time a gene therapy product for any type of hemophilia will be reviewed for marketing authorization by health authorities.