Ultomiris has been approved in the European Union for expanded use to include children + adolescents with paroxysmal nocturnal haemoglobinuria.- Alexion/AstraZeneca

The approval by the European Commission was based on interim results from the Phase III clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients. Ultomiris has an established safety and efficacy profile, offering reduced dosing frequency compared to Soliris (eculizumab), which is the current standard of care in the EU for the treatment of children and adolescents with PNH.

Ultomiris is administered every four or eight weeks (depending on body weight), following a loading dose. The approval follows the recommendation of the Committee for Medicinal Products for Human Use of the European Medicines Agency in July 2021.