Medscape March 25, 2024
The US Food and Drug Administration (FDA) has expanded the indication for the long-acting C5 complement inhibitor, ravulizumab (Ultomiris, Alexion), to include adults with anti–aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).
Ravulizumab is already approved in the United States to treat adults and children aged 1 or older with paroxysmal nocturnal hemoglobinuria or atypical hemolytic uremic syndrome but not Shiga toxin Escherichia coli–related hemolytic uremic syndrome. It’s also approved to treat adults with generalized myasthenia gravis who are anti–acetylcholine receptor Ab+.
The new indication in AQP4 Ab+ NMOSD is based on positive results from the CHAMPION-NMOSD open-label phase 3 trial, which were published last year in Annals of Neurology.
Patients received weight-based intravenous ravulizumab on day 1,...
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