Business
uniQure Announces HOPE-B Clinical Trial Data Published in the New England Journal of Medicine, Demonstrating Durability and Other Benefits of HEMGENIX® (etranacogene dezaparvovec-drlb)
~ Data follow the historic approval of the first gene therapy for hemophilia B, which has been shown in clinical trials to reduce the rate of annual bleeds,
About this update from Uniqure N.v.
[{"type":"text","content":"~ Data follow the historic approval of the first gene therapy for hemophilia B, which has been shown in clinical trials to reduce the rate of annual bleeds, reduce or eliminate the need for prophylactic therapy and generate elevated and sustained factor IX levels for years after a one-time infusion ~ ~ 18-month data support the ongoing benefit of HEMGENIX® ~ ~ HEMGENIX® is approved for adults with hemophilia B in the United States, European Union and European Economic Area ~ LEXINGTON, Mass. and AMSTERDAM, Feb. 23, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that its partner, global biotechnology leader CSL (ASX: CSL) today announced the publication in the New England Journal of Medicine (NEJM) (Vol. 388 No. 8) of results from the pivotal HOPE-B clinical study evaluating the efficacy, durability and safety of HEMGENIX® (etranacogene dezaparvovec-drlb). HEMGENIX® is the first and only gene therapy approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening bleeding, or have repeated, serious spontaneous bleeding episodes. The multi-year clinical development of HEMGENIX® was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment. HEMGENIX® was approved in November 2022 by the U.S. Food and Drug Administration (FDA), and in February 2023 by the European Commission (EC) for the European Union. Results from the HOPE-B trial, the largest gene therapy study in hemophilia B to date, were considered by the authors to demonstrate that HEMGENIX® is superior to routine factor IX prophylaxis in Annualized Bleeding Rate (ABR), factor IX activity, factor IX therapy consumption, factor IX infusion rate, and spontaneous and joint bleeding ABR. Increased factor IX activity was also apparent from week 3 and maintained over 18 months. There were no reported serious adverse events related to treatment with HEMGENIX®. Notably, the results published in NEJM show the ABR of spontaneous bleeding episodes and all joint bleeding episodes decreased by 71% (95% CI: 0.12, 0.71) and 78% (95% CI: 0.10, 0.46), respectively, from lead-in period to post-treatment. Furth...