FDA grants priority review to Sanofi's hemophilia A treatment

The U.S. Food and Drug Administration (FDA) has accepted Sanofi and Sobi’s Biologics License Application (BLA) for efanesoctocog alfa for priority review. The medication is intended for the treatment of hemophilia A, a rare and life-threatening bleeding disorder.

The FDA is expected to make a decision by February 28, 2023. Priority review is given to treatments that have the potential to offer significant improvements in the treatment, diagnosis, or prevention of serious conditions. Efanesoctocog alfa previously received the FDA's Breakthrough Therapy designation in May 2022.

The application is supported by data from the pivotal Phase 3 XTEND-1 study. The study results demonstrated that efanesoctocog alfa delivers high, sustained factor VIII activity levels in the normal to near-normal range for the majority of the week with once-weekly prophylactic dosing. This potentially offers enhanced protection against bleeds.

The study showed clinically meaningful bleed prevention compared to prior factor prophylaxis. Efanesoctocog alfa was well-tolerated, and no inhibitors against factor VIII were detected. The most common treatment-emergent adverse events included headache, joint pain, falls, and back pain.