Breakthrough Therapy designation for factor VIII therapy

Swedish Orphan Biovitrum (Sobi) (STO: SOBI) and French pharma major Sanofi (Euronext: SAN) have been awarded Breakthrough Therapy designation in the USA, for efanesoctocog alfa (BIVV001).

The decision marks the first time a factor VIII therapy has been awarded the status by the US Food and Drug Administration, which has also given the candidate Fast Track designation and Orphan Drug status.

The firms are developing BIVV001 for people with hemophilia A, a rare and life-threatening bleeding disorder, based on data from the Phase III XTEND-1 trial.