KING OF PRUSSIA, PA — CSL (ASX: CSL; USOTC: CSLLY) has unveiled four-year results from its pivotal HOPE-B study, demonstrating the long-term durability and safety of HEMGENIX® (etranacogene dezaparvovec-drlb), the first FDA-approved gene therapy for hemophilia B. The findings, presented at the 18th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD), highlight significant advancements in treating the rare bleeding disorder.
Key results show that 94% of patients treated with a one-time infusion of HEMGENIX were able to eliminate routine factor IX prophylaxis and maintain near-normal factor IX activity levels. The therapy reduced overall annual bleeding rates by approximately 90% by year four, reinforcing its efficacy in providing lasting protection against bleeds. Joint bleeds, a common complication of hemophilia B, saw a dramatic 96% reduction over the same period.
“Hemophilia B can lead to debilitating joint damage and significant pain. These results underscore HEMGENIX’s ability to offer lasting bleed protection with a single treatment, transforming patient outcomes,” said Dr. Steven Pipe, Professor of Pediatrics and Pathology at the University of Michigan.
The study tracked 54 adult male participants with severe or moderately severe hemophilia B, with 51 completing the four-year follow-up. Factor IX activity levels remained consistent over time, averaging 37% at year four, compared to near zero before treatment. HEMGENIX continued to show a favorable safety profile, with no serious adverse events linked to treatment. Treatment-related adverse events, such as increased alanine transaminase (ALT), were recorded in 96% of cases within the first six months and managed effectively with supportive care.
“These data highlight the remarkable impact of HEMGENIX in reducing bleeds and improving the quality of life for patients with hemophilia B,” said Andres Brainsky, Vice President of R&D Hematology at CSL. “We are committed to supporting the hemophilia community by providing long-term data and ensuring patients and healthcare providers have the information they need to make informed treatment decisions.”
HEMGENIX is approved in the U.S., Europe, Canada, and several other regions, with ongoing analyses expected from a post-marketing registry to further validate its long-term efficacy and safety. The therapy’s broad approval for patients with and without AAV5 neutralizing antibodies makes it accessible to a larger population of hemophilia B sufferers.
Looking ahead, CSL aims to continue leading innovation in hemophilia care, leveraging its expertise in gene therapies to transform patient outcomes and redefine treatment possibilities. HEMGENIX stands as a beacon of hope for individuals living with hemophilia B, offering them the potential for greater independence and an improved quality of life.
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