For the first time in Germany, a haemophilia B patient has been treated outside of a trial with the gene therapy developed by CSL Behring and approved in Europe. The rare blood clotting disorder affects over 1,000 people in Germany, around 65 percent of whom have severe or moderate hemophilia. The cause is a genetic deficiency of functional coagulation factor IX, which leads to spontaneous bleeding, joint pain and lengthy infusion therapies.

Gene therapy addresses the cause: A single infusion introduces an intact genetic blueprint into the liver cells, which then produce the coagulation factor. The aim is to alleviate symptoms and replace regular infusions for years. Following EU approval in 2023, the therapy is now part of healthcare in Germany, supported by an agreement on reimbursement with health insurance companies.

Studies show that the therapy increases factor IX activity, reduces bleeding and improves quality of life. Many patients have been able to discontinue their infusions, and model calculations indicate a long-term benefit of up to two decades. The treatment marks a milestone, emphasizes Christian Wieszner, Managing Director of CSL Behring Germany, and sets new standards for success-based reimbursement models.

Christian Schepperle from the Interessengemeinschaft Hämophiler e.V. emphasizes that the therapy is not only medically but also socio-economically significant, as it reduces long-term healthcare costs and improves the quality of life of those affected. This step marks the beginning of a new chapter in haemophilia B care, which could make everyday life easier for patients in the long term.

Read also:

Thalassemia: Gene therapy enables life without blood transfusions – MedLabPortal

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