Hemophilia B is a serious rare inherited disease characterized by insufficient blood clotting. The condition can lead to repeated and sometimes life-threatening episodes of external and internal bleeding. The episodes can cause long-term damage, for example to the joints, and can be fatal if they occur in the brain. The deficient blood clotting results from the lack of functional human Factor IX, or hFIX, a blood clotting factor, as a result of mutations in the relevant gene.
Transformative potential in hemophilia type B.
Current Therapies: Burdensome and Expensive
Treatment of hemophilia B today consists of prophylactic or on-demand protein replacement therapy, in which frequent intravenous administrations of plasma-derived or recombinant hFIX are required to stop or prevent bleeding. Protein replacement therapy is expensive, costing on average more than $600,000 per patient per year in the United States. Such therapy is also burdensome and does not completely prevent bleeding. The lifetime cost of hemophilia B for an adult patient with severe hemophilia B has been shown to be more than $20 million.
uniQure’s Etranacogene Dezaparvovec (AMT-061): Long-Term, Functionally Curative Potential
uniQure’s hemophilia B gene therapy etranacogene dezaparvovec (AMT-061) aims to restore the function of blood clotting on a long-term and potentially curative basis through the delivery of the functional gene for hFIX into the patients’ liver cells. We believe etranacogene dezaparvovec may be the first gene therapy to provide durable, functionally curative benefits to nearly all patients with hemophilia B, with optimized clinical and safety benefits.
uniQure is currently conducting the Phase III HOPE‑B pivotal study of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B.