The FDA has accepted Sanofi and Sobi’s Biologics License Application (BLA) for hemophilia drug efanesoctocog alpha, granting the drug priority review status. The target action date for the FDA decision is February 28, 2023.
The BLA acceptance hinged on data from the XTEND-1 phase 3 study. The results demonstrated clinically meaningful prevention of bleeds and superiority to prior prophylaxis based on patient comparisons.
Efanesoctocog alfa is being examined for use in patients with hemophilia A, a rare genetic disorder in which a patient’s ability to clot becomes impaired due to insufficient factor VIII.
Sanofi and Sobi have been collaborating on the development and commercialization, with the Swedish rare disease retaining the commercialization rights in Europe, North Africa, Russia and most Middle Eastern markets and Sanofi owning the rights in North America and all other regions in the world excluding the Sobi territory.
The drug is an investigational recombinant factor VIII therapy, which builds on fusion technology with added polypeptide to help extend its time in circulation in the drug. It initially received Breakthrough Therapy designation from the FDA in May of this year. Before that, the FDA had granted the drug Orphan Drug designation in August of 2017 and later Fast Track designation in February 2021.