CSL Behring Canada Inc., a division of Australian biotechnology firm CSL Ltd. (CSL.AX, CMXHF.PK), announced on Tuesday that the Canadian Agency for Drugs and Technologies in Health (CADTH) Canadian Drug Expert Committee (CDEC) has recommended public drug plan reimbursement for HEMGENIX (etranacogene dezaparvovec). This gene therapy is intended for eligible adults with hemophilia B (congenital factor IX deficiency) who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
The recommendation is supported by findings from the ongoing Phase III HOPE-B trial. This study demonstrated that a one-time infusion of HEMGENIX in adult males with hemophilia B elevated factor IX activity levels and significantly reduced the annual bleed rate compared to routine factor IX prophylaxis, all while maintaining a favorable safety profile.
Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, leading to insufficient production of factor IX. This protein, primarily produced by the liver, is essential for blood clot formation.
HEMGENIX is the first gene therapy for hemophilia B authorized by Health Canada. It has also received approvals from the U.S. Food and Drug Administration, the European Commission (EC) for the European Union and European Economic Area, the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA), Swissmedic in Switzerland, and has been granted provisional approval in Australia.
