Breakthrough Gene Therapy for Hemophilia B Showcases Transformative Impact in France

Breakthrough Gene Therapy for Hemophilia B Showcases Transformative Impact in France

Elena Moreno
2 min read

Breakthrough Gene Therapy for Hemophilia B Showcases Transformative Impact in France

CSL Behring has recently treated two patients with HEMGENIX, a gene therapy for haemophilia B, at treatment centers in France. Developed by UniQure, HEMGENIX obtained conditional marketing authorization from the European Commission in February. This authorization allows its use in Europe for severe and moderately severe haemophilia B in adult patients without Factor IX inhibitors. The therapy offers a one-time treatment, enabling patients to produce their own Factor IX, ultimately reducing the frequency of bleeding episodes. This milestone was achieved through France's Direct Access scheme, emphasizing the country's commitment to early access for innovative treatments. Dr. Lutz Bonacker, senior vice-president and general manager of CSL Behring Commercial Operations Europe, emphasized the significance of this achievement, highlighting the transformative potential of gene therapy in haemophilia B treatment.

Key Takeaways

  • HEMGENIX, a one-time gene therapy, treats haemophilia B by enabling Factor IX production.
  • CSL Behring treated two patients in France with HEMGENIX after acquiring its commercial rights.
  • The therapy received conditional marketing authorization in the EU and EEA in February 2023.
  • HEMGENIX aims to reduce the risk of bleeding episodes by replacing the missing Factor IX.
  • Direct Access in France allows early access to innovative treatments like HEMGENIX.


The deployment of HEMGENIX in France signifies a pivotal shift in hemophilia B treatment, leveraging the transformative potential of gene therapy. This move positions CSL Behring and UniQure as leaders in innovative healthcare while enhancing France's reputation for medical innovation. Short-term implications include potential investor confidence in biotech stocks, while long-term benefits may entail reduced healthcare costs and enhanced patient outcomes. The success of HEMGENIX could spur further investment in gene therapies, reshaping the genetic medicine landscape.

Did You Know?

    • Details: HEMGENIX, developed by UniQure, is a groundbreaking gene therapy aimed at treating haemophilia B. It utilizes gene transfer to enable patients to produce Factor IX, a crucial protein for blood clotting, ultimately reducing the frequency of bleeding episodes.
  • Conditional Marketing Authorization:
    • Details: This authorization is granted by the European Commission to medicinal products addressing unmet medical needs and showing a positive benefit-risk balance. It enables the early availability of innovative treatments in the EU and EEA, subject to certain conditions such as additional data collection and monitoring.
  • Direct Access Scheme:
    • Details: Implemented in France, this policy allows early access to innovative treatments like HEMGENIX, reflecting the country's commitment to patient welfare and medical innovation advancement.

You May Also Like

This article is submitted by our user under the News Submission Rules and Guidelines. The cover photo is computer generated art for illustrative purposes only; not indicative of factual content. If you believe this article infringes upon copyright rights, please do not hesitate to report it by sending an email to us. Your vigilance and cooperation are invaluable in helping us maintain a respectful and legally compliant community.

Subscribe to our Newsletter

Get the latest in enterprise business and tech with exclusive peeks at our new offerings