The Food and Drug Administration (FDA) has expanded 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 ( Identifier: NCT03406507) that assessed the efficacy and safety of ravulizumab-cwvz in 13 patients 9 to 17 years of age with PNH. Of the 13 patients, 5 had never received treatment 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 once every 8 weeks thereafter for patients weighing at least 20kg, or once every 4 weeks for patients weighing less than 20kg.

Results showed that after 26 weeks, 60% (95% CI, 14.7-94.7) of treatment-naive patients avoided transfusion and 100% (95% CI, 63.1-100) of patients who had received prior eculizumab treatment avoided transfusion. Three out of 5 treatment-naïve patients and 6 out of 8 eculizumab-experienced patients achieved hemoglobin stabilization by week 26, respectively. As for safety, the most common adverse reactions were upper respiratory tract infection and headache. 

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“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 & Blood Disorders Center at Children’s Healthcare of Atlanta, as well as assistant professor of pediatrics at the Emory University School of Medicine. “It’s exciting to finally have an approved medicine for these patients who are diagnosed as children.”

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

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


  1. FDA approves therapy for pediatric patients with serious rare blood disease. [press release]. Silver Spring, MD: US Food and Drug Administration; June 7, 2021. 
  2. 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.
  3. Ultomiris [package insert]. Boston, MA: Alexion Pharmaceuticals; 2021.

This article originally appeared on MPR