Posted by Michael Wonder on 24 Dec 2019
BioMarin submits biologics license application to U.S. FDA for valoctocogene roxaparvovec to treat haemophilia A
23 December 2019 - First marketing application submission in U.S. for gene therapy directed at any type of haemophilia.
BioMarin announced today that the company submitted a biologics license application to the U.S. FDA for its investigational AAV gene therapy, valoctocogene roxaparvovec, for adults with haemophilia A. Subject to completion of the FDA's filing review, BioMarin anticipates the review to commence in February 2020. BioMarin will provide an update in February 2020.
This submission is based on a Phase 3 interim analysis of study participants treated with material from the to-be-commercialised process, and the three-year Phase 1/2 data.
