BioMarin's biologics license application for valoctocogene roxaparvovec accepted for priority review by FDA with review action date of 21 August 2020

BioMarin

20 February 2020 - No advisory committee meeting currently planned to review the application.

BioMarin Pharmaceutical today announced that the U.S. FDA has accepted for priority review the biologics license application to the FDA for its investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with haemophilia A. This acceptance by the FDA marks the first marketing application accepted for a gene therapy product for any type of haemophilia in the United States.

The application is based on a Phase 3 interim analysis of study participants treated with investigational product manufactured by the to-be-commercialized process and three-year Phase 1/2 data. BioMarin has constructed, commissioned, and validated one of the first gene therapy manufacturing facilities of its kind in the world. This award-winning facility is located in Novato, California. Marketing authorisation documentation has been included in the applications, and the facility is ready for inspection to support approval.

Read BioMarin press release

Michael Wonder

Posted by:

Michael Wonder