CSL Limited announced on February 7, 2025, four-year results from the pivotal HOPE-B study for HEMGENIX (etranacogene dezaparvovec-drlb), a gene therapy for adults with hemophilia B. The data confirmed the long-term durability and safety of a one-time infusion, demonstrating sustained clinical benefits.
Through four years, HEMGENIX maintained elevated and sustained factor IX activity levels, with a mean of 37.4 IU/dL at year four. The therapy also achieved an approximately 90% reduction in the mean adjusted annualized bleeding rate for all bleeds compared to lead-in, and 94% of patients remained free of continuous prophylaxis treatment in year four.
No serious adverse events related to HEMGENIX treatment were reported, and the therapy was generally well-tolerated. uniQure N.V. led the multi-year clinical development of HEMGENIX before licensing global rights to CSL, and these positive long-term results validate uniQure's gene therapy platform and its potential for durable therapeutic effects.
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