BioMarin Submits First European Marketing Application for Hemophilia Gene Therapy

January 6, 2020

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.

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