Ultomiris recommended for approval in the EU by CHMP for children and adolescents with paroxysmal nocturnal haemoglobinuria
Opinion based on results from Ultomiris Phase III trial that showed an established efficacy and safety profile with reduced treatment burden for children with PNH and their familiesAlexion’s Ultomiris (ravulizumab) has been recommended for marketing authorisation in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH). The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on interim results from the Phase III clinical trial in children and adolescents