Gene therapy for hemophilia B approved by European Commission

The European Commission has granted conditional marketing authorization (CMA) for CSL’s HEMGENIX (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors.

In the ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds with a single infusion by delivering a functional gene that acts as a blueprint for coagulation Factor IX, a protein important for blood clotting. It is the first approved gene therapy for 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, 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.”

People living with hemophilia B currently require lifelong treatment of intravenous infusions of Factor IX to maintain sufficient levels, which can have a significant impact on their quality of life and wellbeing. According to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), there is “an unmet medical need for new therapeutic approaches that might free patients from the burden of frequent infusions, or episodically at the time of a bleeding event.”

The European Commission’s decision follows the CHMP’s positive opinion in December 2022, based on findings from the pivotal HOPE-B trial, the largest gene therapy trial in hemophilia B to date. These findings showed that hemophilia B patients treated with HEMGENIX demonstrated stable and durable increases in mean Factor IX activity levels (with a mean Factor IX activity of 36.9%) which led to an adjusted annualized bleed rate (ABR) reduction of 64%. Following infusion of HEMGENIX, 96% of patients discontinued routine Factor IX prophylaxis and mean Factor IX consumption was reduced by 97% at 18 months post-treatment, compared to the lead-in period.

The HOPE-B study 24-month analysis continued to show a sustained and durable effect of HEMGENIX. In a clinical setting, the treatment is generally well-tolerated with no serious treatment-related adverse events.

“This approval marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints and internal organs, alleviating the burden of lifelong intravenous infusions of Factor IX products,” said professor Wolfgang Miesbach, head of Coagulation Disorders at the Comprehensive Care Center, University Hospital of Frankfurt. “Data from the HOPE-B study demonstrate the potential of HEMGENIX to remove the need for routine prophylaxis, by providing durable Factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B.”

“At CSL Behring, our promise is simple – to save and improve lives – and we achieve this by living our core values of patient focus, innovation, integrity, collaboration, and superior performance,” commented Lutz Bonacker, senior vice president and general manager of Commercial Operations Europe, CSL Behring. “The approval of HEMGENIX in Europe is a result of that focus and a milestone for the hemophilia B community, and we now need to work to ensure that as many eligible patients across Europe can access this innovative treatment as possible. We are fully committed to working together with payers and other stakeholders to achieve this.

The European Commission has the authority to approve medicines for EU Member States, as well as well as in the EEA countries of Iceland, Norway and Liechtenstein.

The multi-year clinical development of HEMGENIX was led by uniQure and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment. In the United Kingdom, The Medicines and Healthcare products Regulatory Agency (MHRA) is currently reviewing CSL’s submission for HEMGENIX. HEMGENIX was approved by the U.S. Food and Drug Administration in November 2022.

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