The European Commission has approved etranacogene dezaparvovec (Hemgenix), the first gene therapy for severe and moderately severe hemophilia B (congenital factor X1 deficiency).
The results showed that patients with hemophilia B treated with the gene therapy demonstrated stable and durable increases in mean factor IX activity , which led to an adjusted annualized bleeding rate reduction of 64%.
After receiving the gene therapy, 96% of patients discontinued routine factor IX prophylaxis and mean factor IX consumption was reduced by 97% at 18 months post-treatment compared with the lead-in period, the company noted. "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," commented one of the trialists, Professor Wolfgang Miesbach, Head of Coagulation Disorders at the Comprehensive Care Center, University Hospital of Frankfurt, Germany.
This European 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," Miesbach said in the company press release.
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