CSL announced that Health Canada has authorized HEMGENIX® (etranacogene dezaparvovec), the first and only gene therapy for the treatment of hemophilia B. HEMGENIX is indicated for the treatment of adults with hemophilia B who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes. There is no clinical experience of HEMGENIX use in patients with mild or moderate hemophilia B (FIX activity >2%).
The approval is based on results from the ongoing Phase III, open label, single-dose, single-arm, multi-center HOPE-B trial of 54 participants, the largest gene therapy trial in hemophilia B to date. Data from the pivotal trial show that people with hemophilia B treated with a single infusion of HEMGENIX demonstrated significant increases in mean FIX activity levels (as measured by one-stage assay) of 36.9% at 18-months, that were sustained at 36.7% at 24-months post-treatment, compared to the six-month lead in period. Seven to 18 months post-infusion, the mean ABR was reduced by 58% compared to the six-month lead-in period (4.13 to 1.73). Following infusion of HEMGENIX, 96% of patients (52 out of 54) discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy. Of the adverse events reported based on phase 2b and phase 3 trial (Hope-B), most frequently reported adverse drug reactions were ALT elevations, headache, influenza-like illness and AST elevations.
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 the blood to properly clot. Treatments for hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules. Those with hemophilia B may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage, or severe pain as a result of the disease. HEMGENIX allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.
HEMGENIX® was also approved by the FDA and was granted conditional marketing authorization by the European Commission (EC) for the European Union and European Economic Area, and the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA). The multi-year clinical development of HEMGENIX® was led by uniQure (Nasdaq: QURE) and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment.
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