BioMarin Reports Positive Phase 3 Data for Hemophilia Drug

BioMarin Pharmaceutical said its experimental gene therapy reduced bleeding by 85 percent in a global phase 3 study evaluating the treatment valoctocogene roxaparvovec in 134 patients with hemophilia A.

The San Rafael, Calif.-based drugmaker, which has filed for marketing authorization with the European Medicines Agency (EMA), said it expects to receive an opinion from the EMA’s human medicines committee during the first half of this year.

In the U.S., BioMarin plans to meet with the FDA to discuss these latest findings and expects to file a biologics license application in the second quarter of this year.