The U.S. Food and Drug Administration (FDA) has accepted for priority review the Biologics License Application (BLA) for efanesoctocog alfa (BIVV001) for the treatment of hemophilia A, a rare and life-threatening bleeding disorder. The target action date for the FDA decision is February 28, 2023. Sanofi and Sobi® collaborate on the development and commercialization of efanesoctocog alfa.
The BLA is supported by data from the pivotal XTEND-1 Phase 3 study. Results were recently presented at the 30th International Society of Thrombosis and Haemostasis Congress. The data demonstrate a clinically meaningful prevention of bleeds and superiority to prior factor prophylaxis based on an intra-patient comparison. Efanesoctocog alfa was well-tolerated, and inhibitor development to factor VIII was not detected. The most common treatment- emergent adverse events (>5% of participants overall) were headache, arthralgia, fall, and back pain.
The FDA grants priority review to therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Efanesoctocog alfa received Breakthrough Therapy designation from the FDA in May 2022 and it is the first factor VIII therapy to receive this recognition. The FDA also granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.
Regulatory submission in the EU will follow availability of data from the ongoing XTEND- Kids pediatric study, with both events expected in 2023. The European Commission granted efanesoctocog alfa Orphan Drug designation in June 2019.