FDA breakthrough designation to efanesoctocog alfa for haemophilia A

June 2022 Pharma News Nalinee Pathak
Haemophilia - blood disorder abstract.

The Food and Drug Administration (FDA) has granted breakthrough therapy designation to efanesoctocog alfa for the treatment of haemophilia A.

The drug efanesoctocog alfa or BIVV001, Sanofi is a recombinant factor therapy that has been designed to prevent bleeding in haemophilia patients with once-weekly prophylactic dosing.


The decision by FDA is based on the results of the phase III XTEND-1 study. The study’s primary endpoint shows that efanesoctocog alfa usage results in meaningful prevention of bleeding in people with severe haemophilia A. The drug also met its secondary endpoint as it was found to be superior compared to prophylactic factor VIII replacement therapy. 

The efanesoctocog alfa showed acceptable safety and tolerability profile, with the most common adverse events being headache, arthralgia, falls and back pain.

Orphan drug

The data from the XTEND-1 study are expected to be shared at an upcoming meeting this year. Earlier, in 2017, the FDA has designated efanesoctocog alfa orphan drug, and the European commission granted the same status in June 2019.

According to John Reed, MD, PhD, global head of research and development at Sanofi, “The breakthrough therapy designation highlights efanesoctocog alfa’s potential to transform treatment for people with haemophilia A by providing higher protection for a longer duration,”.


Press release from Sanofi