US closer to commercialising first gene therapy for haemophilia

Credit: wacomka

The Biologics License Application for BioMarin Pharmaceutical’s investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with haemophilia A is closer to approval in the US after it was accepted for Priority Review.

This marks the first marketing application accepted for a gene therapy product for any type of haemophilia in the US.

The application is based on a Phase 3 interim analysis of study participants treated with investigational product manufactured by the to-be-commercialised process and three-year Phase 1/2 data.

In Novato, California, BioMarin has constructed, commissioned and validated one of the first gene therapy manufacturing facilities of its kind in the world.

Marketing authorisation documentation has been included in the applications, and the facility is ready for inspection to support approval.

In addition, the FDA has accepted the premarket approval application for an AAV5 total antibody assay intended as a companion diagnostic test for valoctocogene roxaparvovec.

The FDA has granted valoctocogene roxaparvovec Breakthrough Therapy designation.  Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the European Medicines Agency.

For this latest step, the FDA has set an action date of August 21.


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