In February, HEMGENIX® was approved by the European Commission as the first gene therapy for hemophilia B, a rare form of a bleeding disorder. The innovative gene therapy is now also available in Germany and can replace the previous lifelong substitution therapy with coagulation factors in adult patients with severe and moderate hemophilia B and without a history of factor IX inhibitors for a longer period of time.
The new gene therapy treatment with HEMGENIX® offers suitable patients the opportunity to address a previously unmet medical need: 96% of all patients in the pivotal HOPE-B study no longer required routine factor IX prophylaxis after gene therapy infusion, leading to an improvement of quality of life in study participants.
About hemophilia B
Hemophilia B is a life-threatening rare disease. People with this condition are particularly prone to bleeding in the joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Current treatment for moderate to severe hemophilia B includes lifelong infusions of factor IX to briefly correct low levels of coagulation factor.