FDA approves Ultomiris for children and adolescents with paroxysmal nocturnal hemoglobinuria.- Alexion Pharmaceuticals

This approval is based on interim Phase III study results, which showed that Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age. Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis, which can lead to disabling or potentially fatal blood clots. The efficacy and safety of Ultomiris in children and adolescents is consistent with the established profile of Ultomiris in clinical studies involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting. The results of the pediatric study continue to demonstrate Alexion’s commitment to treating complement-mediated diseases, which spans more than 17 clinical trials – including the largest pediatric and adult trials in PNH completed to date – and over 60,000 patient-years of trial and real-world data, across more than 50 countries. Data from the interim analysis will be presented as an e-poster during the European Hematology Association 2021 Virtual Congress and will be made available on the congress website on June 11, 2021 at 9:00 a.m. CEST (3:00 a.m. EDT).