The first haemophilia B patient in Germany has been treated with the one-time gene therapy HEMGENIX at the Hemophilia Treatment Center at Hannover Medical School, CSL Behring Germany has announced.
HEMGENIX® is the first gene therapy of its kind approved in Europe for adults with severe and moderately severe haemophilia B. It is intended for patients without a history of Factor IX inhibitors and addresses the underlying genetic cause of the disorder.
Christian Schepperle, Managing Director of the Interessengemeinschaft Hämophiler e.V. in Germany, welcomed the development: “Each advancement in hemophilia treatment, particularly through innovative therapies like gene therapy, brings us closer to a better and more carefree life.”
He added: “Advanced therapies are not only medically essential but also make socio-economic sense by helping to limit long-term healthcare costs ‒ thus making us more economically and geopolitically independent.”
CSL Behring has introduced HEMGENIX in Germany under a novel success-based reimbursement model. This marks the first time such a model has been used in the country.
Christian Wieszner, Managing Director of CSL Behring Germany, said: “We are proud to offer a gene therapy that not only provides a long-term solution for patients but also sets new standards for outcome-based reimbursement models.”
HEMGENIX® received conditional marketing authorisation from the European Commission in February 2023 after US approval in November 2022. It has since been approved in multiple countries including Canada, the UK, Switzerland and Australia.
Patients in France, Denmark, Austria, Spain and the UK have already received the therapy. New data from the HOPE-B study presented in 2025 shows continued safety and long-term durability following a one-time infusion
