Ultomiris Approved for Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria

The Food and Drug Administration (FDA) has extended the approval of Ultomiris (ravulizumab-cwvz) to include patients 1 month of age and older with paroxysmal nocturnal hemoglobinuria (PNH). Previously, the treatment was only indicated for adults 18 years of age and older.

The approval was based on data from a 26-week, open-label, phase 3 study (ClinicalTrials.gov Identifier: NCT03406507) evaluating the efficacy and safety of ravulizumab-CFS in 13 patients aged 9 to 17 years with PNH. Of the 13 patients, 5 had never been treated with complement inhibitors, while 8 had been treated with eculizumab. Patients received a loading dose on Day 1, followed by maintenance treatment on Day 15, and thereafter once every 8 weeks for patients weighing at least 20 kg, or once every 4 weeks for patients weighing less than 20 kg.

The results showed that after 26 weeks, 60% (95% CI, 14.7-94.7) of treatment-naïve patients avoided transfusion and 100% (95% CI, 63.1-100) of patients who previously treated with eculizumab, avoided transfusion. Three of 5 treatment-naïve patients and 6 of 8 eculizumab-experienced patients achieved hemoglobin stabilization at week 26, respectively. In terms of safety, the most common adverse reactions were upper respiratory tract infection and headache.

“It can take months and sometimes years to get the right 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 & Blood Disorders Center at Children’s Healthcare of Atlanta, as well as assistant professor of pediatrics at Emory University School of Medicine. “It’s exciting to finally have an approved drug for these patients who were diagnosed as children.”

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Due to the potential for serious meningococcal infections, Ultomiris is only available through a restricted program called Ultomiris REMS.

Ultomiris is also indicated for the treatment of adults and pediatric patients 1 month of age and older with atypical haemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy.


FDA approves therapy for pediatric patients with severe rare blood disease. [press release]. Silver Spring, MD: US Food and Drug Administration; June 7, 2021. Alexion announces FDA approval of ULTOMIRIS® (ravulizumab-cwvz) for children and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). [press release]. Boston, MA: Alexion Pharmaceuticals, Inc.; June 7, 2021.Ultomiris [package insert]. Boston, MA: Alexion Pharmaceuticals; 2021.


Anemia Haematological disorders Pediatrics

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