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.
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, often costing up to $500,000 to $775,000 per patient per year in the United States. Such therapy is also burdensome and does not completely prevent bleeding.
uniQure’s hemophilia B gene therapy 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 AMT-061 may be the first gene therapy to provide durable, curative benefits to nearly all patients with hemophilia B, with optimized clinical and safety benefits.
AMT-061 has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and access to the Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency.
In June 2018, uniQure began enrolling patients in the Phase III HOPE-B pivotal study of AMT-061 for the treatment of patients with severe and moderately severe hemophilia B.
Read more about AMT-061 here.
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