The Commission European has granted conditional marketing authorisation for etranacogene dezaparvovec (Hemgenix), the first and only gene therapy for the treatment of severe and moderately severe haemophilia B (congenital factor IX deficiency) in adults without a history of factor IX inhibitors.
The approval means that the product will now be available in all countries of the European Union and the European Economic Area.
The gene therapy was approved in the United States in November 2022.It was launched with a price tag of $3.5 million, making it the most expensive treatment to date.
Treatment involves a one-time infusion of a functional gene that serves as a blueprint for clotting factor IX, a protein important for blood clotting, says the manufacturer, CSL.
People with hemophilia B currently need lifetime treatment with intravenous infusions of factor IX to maintain sufficient levels of hemophilia, which can have a significant impact on their quality of life and well-being, the company explains in the press release.
The approval is based on the results of the pivotal HOPE-B study, a single-arm, open-label study of 54 men who relied on factor IX replacement therapy; The first results of this study were presented at the 2020 annual meeting of the American Society of Hematology.
Results showed that patients with hemophilia B treated with gene therapy saw a stable and lasting increase in mean factor IX activity (with mean factor IX activity of 36.9%), which led to an adjusted reduction in annualized bleeding rate of 64%.
After receiving gene therapy, 96 percent of patients discontinued routine factor IX prophylaxis, and average factor IX consumption was reduced by 97 percent at 18 months after treatment compared to the initial period, the company noted.
«Data from the HOPE-B study demonstrate the potential of Hemgenix to eliminate the need for routine prophylaxis, providing long-lasting factor IX activity, as well as improving bleeding outcomes and quality of life for people with haemophilia B,» commented one of the study’s authors, Professor Wolfgang Miesbach, Head of Bleeding Disorders at the Comprehensive Care Center of Frankfurt University Hospital. in Germany.
The European approval «marks an important step forward in the treatment of hemophilia B, which could be transformative for people debilitated by bleeding in muscles, joints and internal organs, alleviating the burden of lifelong intravenous infusions of factor IX products,» Miesbach said in the company’s press release.
This article was originally published on Medscape.com
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