EC nod for Hemgenix, first gene therapy for hemophilia B

The European Commission has granted conditional marketing authorization (CMA) for Hemgenix (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors.

The approval was granted to Australian biotech CSL Limited (ASX: CSL), which gained rights to the treatment from Dutch gene therapy company uniQure (Nasdaq: QURE). The latter led the multi-year clinical development of Hemgenix. Sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment, at a potential cost of over $2 billion.

In the ongoing clinical trial, Hemgenix reduced the rate of annual bleeds with a single infusion by delivering a functional gene that acts as a blueprint for coagulation Factor IX, a protein important for blood clotting. It is the first approved gene therapy for hemophilia B in the European Union (EU) and European Economic Area (EEA).