Posted on: 8 February 2025, source: HCP live
More than 90% of patients with hemophilia B eliminated factor IX prophylaxis 4 years after treatment with etranacogene dezaparvovec (HEMGENIX) gene therapy. Hemophilia B Gene Therapy Achieves Sustained Benefit Over 4 Years. A one-time infusion of etranacogene dezaparvovec-drlb (HEMGENIX) gene therapy continued to demonstrate long-term sustained efficacy and safety in adults with hemophilia B over 4 years in the pivotal Phase 3 HOPE-B study. Announced by CSL on February 7, 2025, these data, presented at the 18th Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD), showed the gene therapy delivered sustained factor IX activity levels, offered greater bleed protection than prophylactic treatment, and maintained favorable safety over time.