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FDA approves ULTOMIRIS® for treatment of rare blood disorder


The US Food and Drug Administration (FDA) has given approval for the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to treat paroxysmal nocturnal hemogloinuria in children and teenagers.

The US Food and Drug Administration has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) from Alexion Pharmaceuticals, Inc. for the treatment of paroxysmal nocturnal hemogloinuria, a rare blood disorder, in children aged 1 month and older.1 The medication was approved for use in adults in 2018 and is the standard of care for treatment. Ravulizumab-cwvz is a long-acting C5 inhibitor.

The approval was the result of findings from an interim Phase 3 trail that showed that the medication was effective in accomplishing complete C5 complement inhibition through 26 weeks in children and adolescents. During the course of the trial, there were no reported severe adverse events related to the treatment. None of the participants stopped treatment during the initial evaluation period or had breakthrough hemolysis. The findings of the trial indicate that both safe and efficacy in the pediatric population are consistent with that found in adults.

Alexion Pharmaceuticals, Inc. will make the drug immediately available for pediatric patients.


1. Alexion Pharmaceuticals. Alexion announces FDA approval of ULTOMIRIS® (ravulizumab-cwvz) for children and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). Published June 7, 2021. Accessed June 8, 2021. https://ir.alexion.com/news-releases/news-release-details/alexion-announces-fda-approval-ultomirisr-ravulizumab-cwvz

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