Positive Phase III trial results for Hemgenix (etranacogene dezaparvovec-drlb) in adults living with hemophilia B – CSL

CSL announced five-year (60-month) results from the pivotal Phase III HOPE-B study, confirming the long-term durability and safety of a one-time infusion of Hemgenix (etranacogene dezaparvovec-drlb) in adults living with hemophilia B. Published in the New England Journal of Medicine (NEJM) and presented simultaneously at the American Society of Hematology (ASH) Annual Meeting, the data reaffirm Hemgenix’s  consistent performance over time to deliver durable factor IX activity levels, sustained bleed protection compared to prophylaxis treatment, and continued freedom from routine prophylaxis. Hemgenix  remains the only commercially available gene therapy for adults with hemophilia B and can be used in patients with or without AAV5 neutralizing antibodies.

“We are incredibly proud to share the five-year results from the HOPE-B study, which reinforce the lasting impact of HEMGENIX as a one-time treatment option for adults with hemophilia B,” said Deborah Long, MD, FCCP, Senior Vice President and Head, Medical Affairs, CSL. “These results highlight the meaningful difference HEMGENIX can make—helping people experience fewer bleeds compared to prophylaxis treatment and freeing them from the burden of regular ongoing treatment. We remain committed to expanding access to this important treatment.”

Although the five-year data mark the final analysis for the HOPE-B study, participants who consent will continue to be monitored in the IX-TEND 222-3003 extended follow-up study (NCT05962398), which will track patients for up to 15 years post-treatment.

The multi-year clinical development of Hemgenix  was led by uniQure (Nasdaq: QURE) and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialize the treatment. CSL also established a post-marketing registry to generate additional long-term safety, efficacy and durability data.

Hemgenix has received regulatory approval in the United States, Canada, the UK, Switzerland, Australia, Saudi Arabia, Taiwan, South Korea, Singapore, and Hong Kong, and conditional marketing authorization from the European Commission (EC) for the European Union and European Economic Area. To date, more than 75 individuals across eight countries have received Hemgenix  in real-world settings.