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.
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.
Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene mutation, resulting in the absence or deficiency in the production of factor IX, a protein primarily produced by the liver that helps blood clots form during the healing process. Treatments for hemophilia B include prophylactic infusions of factor IX 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. They may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease.
For appropriate patients, etranacogene dezaparvovec allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding. In clinical trials, etranacogene dezaparvovec reduced the rate of annual bleeds and the majority of patients treated discounted use of prophylactic treatment after a one-time infusion.
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.