FDA approves first treatment for children and adolescents with PNH
The U.S. Food and Drug Administration has approved the expanded use of ravulizumab-cwvz (Ultomiris; Alexion Pharmaceuticals) to include children >1 month of age and adolescents with paroxysmal nocturnal hemoglobinuria (PNH), according to a press release. This is the first FDA-approved medicine with children and adolescents with PNH. Rvulizumab-cwvz was approved in 2018 for the treatment of adults with PNH.
Approval is based on interim Phase 3 study results which showed that ravulizumab-cwvz was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age. No treatment-related severe adverse events were reported, and no patients discontinued treatment during the evaluation.
Data from the interim analysis will be presented during the European Hematology Association 2021 Virtual Congress on June 11, 2021.
“PNH can have a profound impact on a child’s development and quality of life. With its established safety and efficacy profile, ULTOMIRIS has the potential to transform the lives of children and adolescents suffering from this devastating rare disease,” said John Orloff, MD, Executive Vice President and Head of Research and Development at Alexion. In the press release. “We are inspired by the bravery and resilience of the children and adolescents, as well as their families, who participated in the study, and we are grateful for their commitment — as well as that of the trial investigators — to advancing the understanding of PNH and disease management in younger people. We also appreciate the sense of urgency shown by regulators in prioritizing reviewing and approving the first treatment in the U.S. for children and adolescents with PNH.”
Read the full press release here.