KING OF PRUSSIA, PA — CSL, a global biotherapeutics leader, recently announced the three-year results from its pivotal HOPE-B study. The findings confirm the long-term durability and safety of HEMGENIX (etranacogene dezaparvovec-drlb), a one-time infusion gene therapy for people living with hemophilia B.
HEMGENIX has the distinction of being the first and only gene therapy approved for adults with hemophilia B who currently use factor IX prophylaxis therapy, or have had life-threatening bleeding, or recurrent serious spontaneous bleeding episodes.
The Phase III HOPE-B trial included 54 male participants with severe or moderately severe hemophilia B, some of whom had pre-existing AAV5 neutralizing antibodies. After three years of follow-up, HEMGENIX demonstrated sustained factor IX activity levels, with mean levels of 38.6 IU/dL at year three post-treatment. Importantly, 94% of patients remained free of continuous prophylactic therapy.
The data also showed a significant reduction in the mean annualized bleeding rate (ABR) for all bleeds by 64% during months 7-36 of the study. This reduction mirrored the study’s primary endpoint. Furthermore, median bleeds per participant decreased and remained stable from the lead-in period through to the third year.
HEMGENIX was generally well-tolerated with no serious adverse events related to the treatment reported. Most adverse events were mild, with the most common being an increase in alanine transaminase (ALT).
In addition to the HOPE-B trial results, two posters on HEMGENIX were presented evaluating its use in patients with comorbidities such as chronic hepatitis C, hepatitis B, and HIV. An indirect treatment comparison of HEMGENIX and factor IX prophylaxis was also published in Haemophilia.
HEMGENIX has received conditional marketing authorization from the European Commission and the United Kingdom’s Medicines and Healthcare products Regulatory Agency, as well as authorization by Health Canada.
The 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. Earlier this year, CSL announced the first person living with hemophilia B had received HEMGENIX. For more information on HEMGENIX, visit www.Hemgenix.com.
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