CSL Behring's Gene Therapy HEMGENIX® (etranacogene dezaparvovec-drlb) Four Years Post-Infusion Data Continue to Show Sustained Efficacy and Safety in Adults with Hemophilia B

KING OF PRUSSIA, Pa., Feb. 7, 2025 /PRNewswire/ -- Global biotechnology leader CSL (ASX:CSL; USOTC:CSLLY) today announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of HEMGENIX^® (etranacogene dezaparvovec-drlb) for adults living with hemophilia B. In an oral presentation at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), data showed that through four years, HEMGENIX continues to deliver elevated and sustained factor IX activity levels, can offer long-term and greater bleed protection compared to prophylactic treatment, can eliminate the need for routine factor IX prophylaxis, and maintains a favorable safety profile. Approved in 2022 by the U.S. Food and Drug Administration (FDA), HEMGENIX is the first gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes. It is also the only approved gene therapy for hemophilia B that can treat adult patients with and without AAV5 neutralizing antibodies thereby providing the potential for a greater number of eligible patients to be treated.