Alexion Pharma (ALXN) Confirms FDA approval for ULTOMIRIS for Children and Adolescents with PNH

Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5 inhibitor that offers immediate, complete and sustained complement inhibition, is now the first and only FDA-approved medicine for children and adolescents with PNH.

“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, M.D., principal investigator for the pediatric clinical trial and pediatric hematologist and oncologist at the Aflac Cancer & Blood Disorders Center at Children’s Healthcare of Atlanta, as well as 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.”

Since its initial approval in 2018, ULTOMIRIS has quickly become the standard of care in the U.S. for the treatment of adults with PNH. PNH is a complement-mediated disease, which means the symptoms and complications are caused by a lack of regulation, or control, of the complement system, an essential part of the immune system. ULTOMIRIS is designed to target the part of the complement system at the site of disease activity (terminal complement), while preserving function of other parts of the immune system to be able to fight common pathogens and infections. ULTOMIRIS reduces red blood cell destruction in the blood vessels, also known as intravascular hemolysis, and thrombosis (blood clot) risk by providing immediate, complete, and sustained terminal complement inhibition.

“This expanded approval is a significant step forward for the PNH community as we work to elevate awareness of this rare disease in children and adolescents and ensure patients, both pediatric and adult, have meaningful treatment options available,” said Janice Frey-Angel, Chief Executive Officer and Executive Director of the Aplastic Anemia and Myelodysplastic Syndrome International Foundation (AAMDSIF). “PNH can have significant physical, emotional and/or psychological impacts on families, and we are pleased there is now an approved medicine for the younger members of our community and the families who care for them.”

This approval is based on interim Phase 3 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).

“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, M.D., Executive Vice President and Head of Research and Development at Alexion. “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.”

Alexion plans to make ULTOMIRIS available to pediatric patients in the U.S. immediately. A regulatory filing for ULTOMIRIS in pediatric patients with PNH is under review in the European Union (EU).

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