The FDA granted Sanofi and Sobi’s investigational blood-clotting drug efanesoctocog alfa a Breakthrough Therapy designation for treatment of patients with hemophilia A.
Efanesoctocog alfa is a novel recombinant factor VIII therapy designed to extend protection from bleeds with a once-weekly prophylactic dose.
The breakthrough designation was supported by phase 3 data showing that the treatment helped prevent bleeding in individuals with the rare, life-threatening genetic bleeding disorder over a 52-week period.
The FDA granted efanesoctocog alfa Orphan Drug designation in August 2017 and Fast Track designation in February 2021.
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