On Jan. 15, 2024, CSL announced that Swissmedic had authorised HEMGENIX^® (etranacogene dezaparvovec), the first and currently only gene therapy for the treatment of male adults aged >18 years with severe or moderately severe hemophilia B (congenital Factor IX deficiency). HEMGENIX^® is a one-time, single-dose treatment option that allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.

The approval is based on results from the ongoing Phase III HOPE-B trial, the largest gene therapy trial in hemophilia B to date. Data from the pivotal trial show that HEMGENIX^® reduces the rate of annual bleeding by delivering a functional gene that acts as a blueprint for the production of coagulation factor IX in the liver, a protein that is essential for blood clotting.

HEMGENIX^® has also been approved by the U.S. Food and Drug Administration and authorised by Health Canada, and was granted conditional marketing authorization by the European Commission (EC) for the European Union and European Economic Area, and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).