Hemophilia B

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.

In November 2022, the U.S. Food and Drug Administration (FDA) approved HEMGENIX^® (etranacogene dezaparvovec), the world’s gene therapy for hemophilia B. This is uniQure’s second internally-developed gene therapy to achieve approval and the world’s first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development, as well as the tireless dedication of our employees, clinicians, patients and their families.

In February 2023, HEMGENIX received conditional marketing authorization (CMA) from the European Commission, making it the first and only one-time gene therapy for hemophilia in the EU.

Etranacogene dezaparvovec is is a one-time gene therapy for the treatment of adults with hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

uniQure led the research and clinical development of etranacogene dezaparvovec, which included three clinical trials across 34 global sites and involving 67 adults with hemophilia B.

Treatment of hemophilia B today typically 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 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 has the potential to provide durable, functionally curative benefits to nearly all patients with hemophilia B, with optimized clinical and safety benefits.

In October 2023 uniQure, together with our commercial partner CSL, received the 2023 Prix Galien USA Award in the category of Best Product for Rare/Orphan Diseases for HEMGENIX, the world’s first gene therapy for hemophilia B. The Prix Galien is regarded worldwide as the equivalent of the Nobel Prize for the life science industry. This is a tremendous honor for uniQure and an important recognition of uniQure’s groundbreaking efforts and innovation in the field.