The EMA has accepted BioMarin’s marketing application for valoctocogene roxaparvovec, its investigational gene therapy to treat severe hemophilia A in adults.
The submission is the first in Europe for a gene therapy product for any type of hemophilia.
The submission was based on interim analysis of an ongoing phase 3 study with material from the to-be-commercialized process and updated three-year phase 1/2 data.
The FDA granted valoctocogene roxaparvovec breakthrough therapy designation. It also received and orphan drug designation from the FDA and the EMA.