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

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

The comprehensive approval was supported by interim findings from a phase 3 study, which showed that ravulizumab-CFS has the ability to achieve complete inhibition of the C5 complement for 26 weeks in children and adolescents up to 18 years of age.

Ravulizumab-CFS was also not associated with treatment-related serious adverse events, and none of the patients discontinued treatment during the primary evaluation period, nor was there a hemolysis breakthrough.

“It can take months and sometimes years to get an accurate 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 Emory University School of Medicine. “Managing the disease can be extremely stressful for these children and their families, who often miss school and work for infusions, blood transfusions and medical appointments. It is exciting to finally have an approved drug for these patients diagnosed as children. ”

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

Data from the interim analysis will be presented at 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 change the lives of children and adolescents suffering from this devastating rare disease,” said John Orloff, MD, executive vice president and chief of research and development at Alexion, the developer of ravulizumab. . -cwvz. “We are inspired by the courage 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 study researchers – to advance the understanding of PNH and improve disease management in younger people We also appreciate the sense of urgency shown by regulators in prioritizing the review and approval of the first treatment in the [United States] for children and adolescents with PNH.”

Reference

Alexion announces FDA approval of ULTOMIRIS® (ravulizumab-cwvz) for children and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). news item. Alexie. June 7, 2021. Accessible on June 7, 2021. https://bit.ly/3gk603E

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