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First gene therapy for hemophilia B approved in the EU

The European Commission has granted conditional marketing authorization (CMA) for CSL’s HEMGENIX, the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B 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.”

The HOPE-B trial

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%.1 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, reports the company.

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.

Photo: iStock

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