Sanofi's Efdoralprin Bags Orphan Drug Designation from European Medicines Agency

01:29 AM EST, 12/17/2025 (MT Newswires) -- Sanofi's (SAN.PA) efdoralprin alfa secured orphan designation from the European Medicines Agency for the potential treatment of alpha-1 antitrypsin deficiency-related emphysema, a rare respiratory condition. The designation is awarded to investigational therapies targeting rare, life-threatening conditions affecting no more than five in 10,000 people in the European Union, according to a Wednesday release. In the mid-stage ElevAATe study, efdoralprin alfa met the primary and key secondary endpoints after working better than the current standard of care in improving the alpha-1 antitrypsin levels of patients. Efdoralprin alfa was administered every three or four weeks in ElevAATe. The French pharmaceutical group previously received fast track and orphan drug designations for efdoralprin alfa in the US.