CSL’s Hemgenix Wins First Gene Therapy Approval For Hemophilia In Europe

The European Commission (EC) has given conditional approval to CSL’s Hemgenix (etranacogene dezaparvovec), making it the first approved gene therapy for the treatment of severe and moderately severe hemophilia B in the European Union (EU) and European Economic Area (EEA).

“The approval of Hemgenix in Europe is the essence of great science delivering a medicine that we believe can transform the treatment paradigm for both people living with hemophilia B and the healthcare professionals who treat them,” said Dr. Bill Mezzanotte, Head of Research & Development and Chief Medical Officer, CSL.

Hemgenix is the result of a 2020 licensing collaboration between CSL Behring and uniQure. Under the terms of the agreement, uniQure received a $450 million upfront payment from CSL in May 2021 and could potentially receive up to $1.6 billion in milestones as well as tiered double-digit royalties up to the “low-twenties”.

“Hemgenix, and our partnership with uniQure, underscore CSL’s promise to pursue, develop and deliver disruptive innovations when patients can benefit, particularly in disease states we know well like hemophilia B,” said Dr. Bill. 

Reducing Annual Bleeds and Discontinuing Factor IX Prophylaxis

Hemophilia B is caused by an inherited deficiency in clotting Factor IX, which leads to spontaneous bleeding episodes in the body. People living with this condition require lifelong treatment of intravenous infusions of Factor IX, but Hemgenix offers them a chance to be free. The one-time treatment is designed to deliver a functional gene that restores the function of Factor IX. 

In an ongoing clinical trial called Hope-B, a single infusion of Hemgenix reduced the rate of annual bleeds by 54% during months 7-18. That number went up to 64% six months later. 96% of the 54 treated patients were able to discontinue Factor IX prophylaxis. The 24-month analysis in Hope-B continues to show a sustained and durable effect of Hemgenix. 

No serious adverse reactions to Hemgenix were identified in Hope-B; common reported side effects include liver enzyme elevations, flu-like symptoms, fatigue, and nausea. 

Hemgenix obtained US FDA approval in November 2022. In the UK, the Medicines and Healthcare products Regulatory Agency (MHRA) is currently receiving CSL’s submission for Hemgenix.

Author

Joy Lin

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