Posted by Michael Wonder on 22 Nov 2019
BioMarin submits marketing authorisation application to EMA for valoctocogene roxaparvovec to treat severe haemophilia A
21 November 2019 - First marketing application submission for gene therapy directed at any type of haemophilia.
BioMarin announced today that the company submitted a marketing authorisation application to the EMA for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe haemophilia A. Subject to completion of EMA's validation check, BioMarin anticipates the start of the review to commence in January 2020 under accelerated assessment. BioMarin will provide an update in January 2020.
The company remains on track to submit a biologics license application to the U.S. FDA by the end of the year. The FDA has granted valoctocogene roxaparvovec breakthrough therapy designation. Valoctocogene roxaparvovec has orphan drug designation from the FDA and the EMA.
