FDA grants priority review to efanesoctocog alfa for people with haemophilia A and provides PDUFA date

The target action date for the FDA decision is 28 February 2023. Sobi and Sanofi collaborate on the development of efanesoctocog alfa.

“Factor therapy remains a cornerstone of haemophilia treatment, but innovation has been needed in this area to address challenges related to bleed protection and cumbersome treatment regimens,” said Steve Pipe, MD, Professor and Director of Paediatric Haemophilia and Coagulation Disorders Program, University of Michigan. “If approved, efanesoctocog alfa can deliver close to normal factor activity levels for the majority of the week, potentially offering a new tier of protection. Such therapeutic benefits would represent important advances in unmet medical needs for people with haemophilia A and may transform the prophylactic treatment landscape.”

The BLA is supported by data from the pivotal XTEND-1 phase III 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 ( greater than 5% of participants overall) were headache, arthralgia, fall, and back pain.

Regulatory submission in the EU will follow availability of data from the ongoing XTEND-Kids paediatric study, with both events expected in 2023. The European Commission granted efanesoctocog alfa Orphan Drug designation in June 2019.