FDA approves Sanofi long-lasting hemophilia drug

Feb. 24, 2023

French drugmaker Sanofi and partner Sobi announced this week that the U.S. FDA has approved its long-lasting hemophilia A treatment, Altuviiio.

The drug, which was granted priority review last summer, was jointly developed by Sanofi and Sweden-based Sobi, an international rare disease biopharma.

Altuviiio, previously referred to as efanesoctocog alfa, is a first-in-class therapeutic that provides high-sustained factor III replacement designed to extend protection from bleeds with once-weekly dosing for hemophilia A patients of all ages. The drug’s mechanism has been shown to break through the von Willebrand factor ceiling, a blood disorder that causes half-life limitation on other factor VII therapies. 

The agency’s approval hinged on data from the XTEND-1 phase 3 studies, in which the drug provided superior bleeding prevention to restudy prophylaxis, and a normal to near normal factor VIII activity, as well as general improvements in patient’s pain and joint health. 

Sanofi has development and commercialization rights in North America and all other regions in the world excluding Sobi territory.