Hemgenix: The First Gene Therapy for Hemophilia B

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The FDA has approved Hemgenix as the first gene therapy for treating hemophilia B. Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps blood clots form. Current treatments for moderate to severe hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor. Hemgenix is a gene therapy that has been shown to be effective in reducing the symptoms of hemophilia B. The FDA approval is based on results from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. Results from the study demonstrated that HEMGENIX allowed patients to produce mean factor IX activity of 39% at 6 months and 36.7% at 24 months post infusion. 7 to 18 months post-infusion, the mean adjusted annualized bleeding rate (ABR) for all bleeds was reduced by 54% compared to the six-month lead-in period on factor IX prophylactic replacement therapy. In addition, 94% of patients treated with HEMGENIX discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy. uniQure conducted the research and clinical development for the product, which included three clinical trials across 34 global sites and involving 67 adults with hemophilia B. In May 2021, uniQure and CSL completed a licensing transaction providing CSL Behring with exclusive rights to commercialize and continue clinical development of HEMGENIX globally. CSL Behring plans to make Hemgenix available as soon as possible.