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Data Published in the New England Journal of Medicine Confirm the Long-term Durability and Safety of HEMGENIX® (etranacogene dezaparvovec-drlb) Over Five Years
Global biopharma leader CSL (ASX:CSL; USOTC:CSLLY) today announced five-year (60-month) results from the pivotal Phase 3 HOPE-B study, confirming the long-term durability and safety of a one-time infusion of HEMGENIX® (etranacogene dezaparvovec-drlb) in adults living with hemophilia B. Published in the New England Journal of Medicine (NEJM) and presented simultaneously at the American Society of Hematology (ASH) Annual Meeting, the data reaffirm HEMGENIX's consistent performance over time to del
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[{"type":"text","content":"- 94% of patients (51 of 54) remained free from the burden of continuous prophylaxis treatment through five years following a single infusion of HEMGENIX, demonstrating sustained therapeutic benefit- At year five, mean factor IX activity levels remained strong at 36.1% and HEMGENIX continued to demonstrate a favorable safety profile, reinforcing its durable efficacy- More than 75 individuals across eight countries have received HEMGENIX in real-world settings, reflecting growing global adoption","length":511,"tagName":"p"},{"type":"text","content":"KING OF PRUSSIA, Pa., Dec. 7, 2025 /PRNewswire/ -- Global biopharma leader CSL (ASX:CSL; USOTC:CSLLY) today announced five-year (60-month) results from the pivotal Phase 3 HOPE-B study, confirming the long-term durability and safety of a one-time infusion of HEMGENIX® (etranacogene dezaparvovec-drlb) in adults living with hemophilia B. Published in the New England Journal of Medicine (NEJM) and presented simultaneously at the American Society of Hematology (ASH) Annual Meeting, the data reaffirm HEMGENIX's consistent performance over time to deliver durable factor IX activity levels, sustained bleed protection compared to prophylaxis treatment, and continued freedom from routine prophylaxis. HEMGENIX remains the only commercially available gene therapy for adults with hemophilia B and can be used in patients with or without AAV5 neutralizing antibodies.","length":879,"tagName":"p"},{"type":"image","alt":"CSL Logo","displaySize":"","headline":null,"caption":"CSL Logo","className":"","disableSlideshowImg":false,"size":{"original":{"width":400,"height":301,"url":"https://media.zenfs.com/en/prnewswire.com/85f20c566043a2f45a8f50dbec1e5727"},"resized":{"url":"https://s.yimg.com/ny/api/res/1.2/KDRdUCrG.qvO3Z8VT_ocfQ--/YXBwaWQ9aGlnaGxhbmRlcjt3PTcwNTtoPTUzMTtjZj13ZWJw/https://media.zenfs.com/en/prnewswire.com/85f20c566043a2f45a8f50dbec1e5727","width":400,"height":301}},"href":"https://mma.prnewswire.com/media/341673/CSL_logo.html","hrefExternal":true,"rel":"nofollow"},{"type":"text","content":""The five-year HOPE-B results mark a pivotal milestone for gene therapy, providing clear, long-term data of the ability of HEMGENIX to potentially transform care for adults with hemophilia B," said Steven Pipe, MD, Professor of Pediatrics and Pathology, Hemophilia and Co...