The US Food and Drug Administration (FDA) approved ravulizumab-cwvz (Ultomiris®) by Alexion for the treatment of adults with generalized myasthenia gravis (MG) who are positive for the anti-acetylcholine receptor (AChR) antibody. This represents 80% of all patients with MG.
“With the approval of ULTOMIRIS, we’re excited that MG patients now have another option to consider as part of their personalized treatment strategies that may offer more convenience and improve muscle weakness,” said Samantha Masterson, chief executive officer of the Myasthenia Gravis Foundation of America, in a press release.
The FDA’s decision is based on the results of a phase 3 clinical trial called CHAMPION-MG, which were recently published in the New England Journal of Medicine Evidence and presented at the 2022 American Academy of Neurology Annual Meeting.
The double-blind, placebo-controlled, multicenter 26-week trial assessed the safety and efficacy of the treatment in 175 adults with generalized MG. Participants either received ravulizumab-cwvz or a placebo for 26 weeks.
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The primary endpoint was the change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at week 26. Secondary endpoints included change from baseline in the Quantitative Myasthenia Gravis total score and the Revised 15 Component Myasthenia Gravis Quality of Life at week 26.
The results showed that ravulizumab-cwvz led to an improvement in MG-ADL total score compared to placebo.
Patients who completed the randomized control period of the study can continue into an open-label extension period that is currently further evaluating the safety and efficacy of ravulizumab-cwvz. The estimated completion date of the trial is December 31, 2023.
Ravulizumab-cwvz is an intravenous long-acting C5 complement inhibitor. It is currently also under review in the European Union and in Japan.
ULTOMIRIS® (ravulizumab-cwvz) approved in the US for adults with generalized myasthenia gravis. News release. Alexion; April 28, 2022.
Safety and efficacy study of ravulizumab in adults with generalized myasthenia gravis. US National Library of Medicine. Updated March 15, 2022. Accessed April 28, 2022.