EU approves Hemgenix for hemophilia B
CSL announced that the European Commission has granted conditional marketing authorization (CMA) for 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 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.
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