UniQure's HEMGENIX Gene Therapy Treats Haemophilia B Patients in France

UniQure's HEMGENIX Gene Therapy Treats Haemophilia B Patients in France

Elena Kuznetsova
2 min read

Breakthrough in Gene Therapy for Haemophilia B Treatment

CSL Behring has successfully administered HEMGENIX, a groundbreaking gene therapy for haemophilia B, to two patients at medical facilities in France. The innovative therapy, developed by UniQure, obtained conditional marketing authorization from the European Commission in February, authorizing its application in adult patients in Europe suffering from severe and moderately severe haemophilia B without Factor IX inhibitors. This pioneering treatment presents a one-time solution, empowering patients to generate their own Factor IX, potentially mitigating the frequency of bleeding episodes. Previously, managing haemophilia B often necessitated onerous and frequent interventions. Dr. Lutz Bonacker, the senior vice-president and general manager at CSL Behring, emphasized the momentous nature of this achievement, highlighting the transformative impact of gene therapy on the lives of individuals with haemophilia B. The introduction of this treatment under a Direct Access scheme in France signifies a remarkable progression towards providing prompt access to innovative treatments for patients.

Key Takeaways

  • HEMGENIX is a one-time gene therapy designed for haemophilia B patients.
  • It facilitates the production of patients' own Factor IX.
  • Conditional marketing authorization in Europe caters to severe and moderately severe cases without Factor IX inhibitors.
  • The first two patients in France were treated under the Direct Access initiative.
  • The therapy aims to alleviate the challenges of frequent treatments and bleeding episodes.


The successful implementation of HEMGENIX presents substantial financial and reputational advantages for both CSL Behring and UniQure. Furthermore, France's healthcare system and haemophilia B patients stand to benefit from reduced treatment frequencies and associated costs. The conditional approval from the European Commission expedites access to innovative therapies, influencing future regulatory strategies. In the short term, HEMGENIX alleviates the patients' burdens while potentially reshaping the global management of haemophilia B in the long run, thereby impacting pharmaceutical investments and research trajectories.

Did You Know?

  • Gene Therapy:
    • Gene therapy involves the modification of a patient's genes to address or prevent diseases. In the context of HEMGENIX, it is harnessed to treat haemophilia B by introducing a functional copy of the gene responsible for producing Factor IX, a pivotal protein for blood clotting.
  • Haemophilia B:
    • Also known as Christmas disease, haemophilia B is a genetic condition characterized by deficient or defective blood clotting factor IX. This ailment leads to extended bleeding episodes, potentially resulting in life-threatening situations if not appropriately managed.
  • Direct Access Scheme:
    • The Direct Access scheme enables patients to directly receive cutting-edge treatments from healthcare providers without waiting for full regulatory approval. This mechanism plays a crucial role in providing timely access to potentially life-saving treatments, particularly for conditions like haemophilia B, where innovative therapies can significantly enhance patient outcomes.

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