FDA Grants Expanded Approval of Ravulizumab in Paroxysmal Nocturnal Hemoglobinuria for Pediatric Patients

The FDA has granted the expanded use of ravulizumab-cwvz to include the treatment of children aged 1 month and older and adolescents with paroxysmal nocturnal hemoglobinuria.

The FDA has granted the expanded use of ravulizumab-cwvz (Ultomiris) to include the treatment of children aged 1 month and older and adolescents with paroxysmal nocturnal hemoglobinuria, according to a press release from Alexion Pharmaceuticals, Inc.

The expanded approval was supported by interim findings from a phase 3 trial, which showed that ravulizumab-cwvz has the ability to achieve complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years old.

Ravulizumab-cwvz also was not associated with any treatment-related severe adverse events, and no patients discontinued therapy during the primary evaluation period, nor did they have any breakthrough hemolysis.

“It can take months, and sometimes years, to receive a correct diagnosis for PNH — a chronic, progressive and potentially life-threatening rare disease – which can be an overwhelming experience for children and their families,” said Satheesh Chonat, MD, principal investigator for the pediatric clinical trial and pediatric hematologist and oncologist at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta, and assistant professor of pediatrics at the Emory University School of Medicine. “Managing the disease can be extremely burdensome for these children and their families, who often miss school and work for infusions, blood transfusions, and medical appointments. It’s exciting to finally have an approved medicine for these patients who are diagnosed as children.”

Ravulizumab-cwvz is a long-acting C5 inhibitor that is designed to provide complement inhibition. The decision marks the first and only FDA-approved treatment for this pediatric PNH population. The agent was previously granted FDA approval in December 2018 for the treatment of adult patients with PNH, which is a rare blood disorder that leads to hemolysis.

Data from the interim analysis will be presented during the 2021 European Hematology Association 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, the developer of ravulizumab-cwvz. “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 [United States] for children and adolescents with PNH.”


Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH). News release. Alexion. June 7, 2021. Accessed June 7, 2021. https://bit.ly/3gk603E