CSL Behring Germany has announced that the first haemophilia B patient in Germany was treated with the gene therapy Hemgenix (etranacogene dezaparvovec) at the Hemophilia Treatment Center at Hannover Medical School.

Hemgenix is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors1.

“Each advancement in haemophilia treatment, particularly through innovative therapies like gene therapy, brings us closer to a better and more carefree life,” explains Christian Schepperle, Managing Director of the Interessengemeinschaft Hämophiler in Germany. “Our objectives in haemophilia treatment align closely with economic considerations. 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.”

Hemgenix is now available to patients in Germany under an innovative, success-based reimbursement model being implemented for the first time in the country.

“The treatment of the first patient in Germany with Hemgenix marks a milestone in haemophilia B therapy. 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. The agreement with the GVK-Spitzenverband addresses critical challenges such as the long-term efficacy of this one-time therapy and ensures that reimbursement is tied to the individual treatment success of each patient. Additionally, it guarantees that the treatment costs for Hemgenix remain cost-neutral for statutory health insurance (GKV),” explains Christian Wieszner, Managing Director of CSL Behring Germany. 

International approvals  

Hemgenix was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the US Food and Drug Administration (FDA) in November 2022. Hemgenix has also received regulatory approval in Canada, the UK, Switzerland, Australia, Saudi Arabia, Taiwan, South Korea and Hong Kong. 

Following these approvals, CSL Behring is working with relevant stakeholders to ensure eligible patients have access to Hemgenix across Europe and beyond, building on milestone access decisions in Germany, Denmark, Switzerland, Spain, the UK (including Scotland), and Austria. Patients in France, Denmark, Austria, Spain and the UK have already experienced treatment with Hemgenix. 

Additionally, new four-year data from the HOPE-B study presented at the Annual Congress of the European Association for Haemophilia and Allied Disorders 2025 showed that a one-time infusion of HEMGENIX continues to offer long-term durability and safety2. 

“CSL Behring is dedicated to providing opportunities for people with rare genetic bleeding disorders to better manage the impact of their condition on their daily lives. Treatments such as gene therapy have the potential to enable people living with haemophilia B to move towards a haemophilia-free mind,” concluded Wieszner.

Megan Thomas, Multimedia Editor, DDW

References 

  1. Genezen MA, Inc. CSL Behring GmbH. Hemgenix® (etranacogene dezaparvovec): Summary of Product Characteristics [online]. Available at: https://www.ema.europa.eu/en/documents/product-information/hemgenix-epar-product-information_en.pdf [Last accessed: June 2025].
  2. Leebeek FWG, von Drygalski A, et al. The phase 3 HOPE-B trial shows 4-year durability of sustained near-normal FIX activity, bleed protection and favourable safety in adults with severe or moderately severe haemophilia B. EAHAD 2025. EAHAD25-ABS-1255.