Germany Welcomes First Haemophilia B Gene Therapy Patient
Germany has reached a landmark moment in the treatment of haemophilia B, as the first patient in the country has now received the revolutionary one-time gene therapy, HEMGENIX®.
Administered at the renowned Hemophilia Treatment Center at Hannover Medical School, the therapy offers hope of a longer-term, potentially life-altering solution for adults with severe and moderately severe haemophilia B.
The development was announced by CSL Behring Germany, marking a significant step forward in the evolution of haemophilia care. HEMGENIX® is the first gene therapy of its kind to gain approval in Europe, and is specifically intended for patients without a history of Factor IX inhibitors.
Unlike conventional treatments that require lifelong infusions, HEMGENIX® addresses the root genetic cause of the disorder through a one-time intravenous infusion, potentially removing the need for frequent treatment and offering sustained improvements in clotting function.
A Step Towards a More Independent Life
The therapy’s introduction has been warmly received by Germany’s haemophilia community. The Managing Director of the Interessengemeinschaft Hämophiler e.V., one of the country’s leading haemophilia advocacy groups, praised the development, calling it a vital step towards a better and more carefree life for those living with the condition.
What’s more, they highlighted the importance of innovative treatments not only for their clinical impact but also for their wider social and economic benefits.
They also emphasised that advanced therapies like gene therapy don’t just help patients medically, they also make sense from a socio-economic perspective, reducing long-term healthcare costs and contributing to greater economic and geopolitical independence.
A First in Reimbursement Innovation
Equally groundbreaking is how CSL Behring has introduced HEMGENIX® in Germany. The therapy will be made available through a novel success-based reimbursement model – a first for the country.
Under this model, the cost of the treatment is tied to the achievement of specific health outcomes, aligning payment with performance and reinforcing the long-term value that gene therapy is expected to deliver.
The Managing Director of CSL Behring Germany described this as a proud moment for the company and the healthcare sector as a whole. They 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.
A Growing Global Footprint
HEMGENIX® received conditional marketing authorisation from the European Commission in February 2023, following its initial approval in the United States in November 2022. Since then, it has been approved in a growing number of countries, including Canada, the United Kingdom, Switzerland, and Australia.
Across Europe, patients in France, Denmark, Austria, Spain, and the UK have already received the therapy.
The latest data from the ongoing HOPE-B clinical study, shared in 2025, continues to support the safety and long-term durability of HEMGENIX® following a single infusion. This strengthens the case for its broader adoption and long-term use as a transformative therapy for suitable haemophilia B patients.
A Transformative Future for Haemophilia B
With this first patient now treated in Germany, HEMGENIX® is not only providing immediate hope to individuals and families living with haemophilia B, but is also helping reshape the broader landscape of rare disease care.
By addressing the condition at a genetic level and coupling it with an innovative outcomes-based approach to access, this therapy represents a new era – one where advanced science, patient outcomes, and healthcare sustainability go hand in hand.
As more patients across Germany and beyond become eligible for this therapy, the promise of a more manageable and independent life with haemophilia B moves closer to becoming a widespread reality.
News Credits: First German haemophilia B patient receives gene therapy HEMGENI
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