ASH 2023: CSL's Hemgenix provides long-term benefits for hemophilia B patients

Australian biotech CSL Limited (ASX: CSL) has announced the three-year results from the pivotal HOPE-B study confirming continued long-term durability and safety of Hemgenix (etranacogene dezaparvovec-drlb) following a one-time infusion in people living with hemophilia B.

The data showing that a one-time infusion of Hemgenix offers elevated and sustained factor IX activity levels for years were presented in an oral presentation at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition.

Hemgenix is the first and only gene therapy for the treatment approved by the US Food and Drug Administration (FDA) for 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 approved in the European Union and the UK.