Sobi® announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has issued a positive opinion recommending approval of efanesoctocog alfa, for the treatment and prevention of bleeds and perioperative prophylaxis in haemophilia A. Efanesoctocog alfa is a once-weekly and high-sustained factor VIII replacement therapy for patients of all ages and any disease severity. Efanesoctocog alfa provides children, adolescents and adults with normal to near-normal factor VIII activity levels (above 40%) for a significant part of the week with once-weekly dosing, reaching trough levels of 15% in adults and adolescents at day 7. This results in significantly improved protection from bleeds compared to existing factor VIII prophylaxis. The CHMP positive opinion will now be submitted to the European Commission for a marketing authorisation decision.

The recommendation from the CHMP is based on the results from the pivotal phase 3 studies: XTEND-1 in adults and adolescents and XTEND-Kids in children, which evaluated the efficacy and safety of efanesoctocog alfa. These studies demonstrated that once-weekly efanesoctocog alfa prophylaxis (50 IU/kg) provided significant bleed protection (mean ABR <1 and 80-88% of patients free from spontaneous bleeds) in people with severe haemophilia A of any age. The data also showed substantial improvement in joint health, physical health, pain and overall quality of life when comparing week 52 and baseline measurements.1 No factor VIII inhibitors were observed in the efanesoctocog alfa clinical program.

Efanesoctocog alfa was first approved in the US in February 2023 by the US Food and Drug Administration (FDA). The FDA previously granted efanesoctocog alfa Breakthrough Therapy designation in May 2022 ? the first factor VIII therapy to receive this designation, Fast Track designation in February 2021, and Orphan Drug designation in 2017.