Treatment With Ravulizumab-cwvz Shows Long-Term Improvements in Generalized Myasthenia Gravis


Ravulizumab-cwvz (Ultomiris) produced statistically significant improvements in measures of functional activity, muscle strength, and quality of life in patients with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.

Treatment with ravulizumab-cwvz (Ultomiris) showed long-term efficacy in adult patients with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG) in a recent trial.

Results from the open-label extension of the phase 3 CHAMPION-MGtrial showed ravulizumab-cwvz was well tolerated and produced statistically significant improvements from baseline in measures of functional activity, muscle strength, and quality of life at 60 weeks of the open label extension, including Myasthenia Gravis-Activities of Daily Living total score (-4.0 [95% CI -4.8, -3.1], p<0.0001).

Ravulizumab-cwvz, the first and only long-acting C5 complement inhibitor, hinders the C5 protein in the terminal complement cascade of the immune system, which can over-respond when activated in an uncontrolled manner, causing the body to attack its own healthy cells. The drug is administered intravenously every 8 weeks in adult patients after an initial loading dose.

Credit: AstraZeneca

gMG—a rare, chronic autoimmune neuromuscular disease that can cause loss of muscle function and severe weakness—can set in at any age, most commonly occurring in women prior to 40 years of age and in men over 60 years of age. Initial symptoms may include slurred speech, double vision, droopy eyelids, and lack of balance, which can frequently lead to disabling fatigue, slurred speech, difficulty swallowing and eating, double or blurred vision, immobility, shortness of breath, and respiratory failure.

“gMG is a complex, devastating disease, disrupting many aspects of daily living, and helping patients improve muscle strength and function should be essential to any treatment plan,” said CHAMPION-MG lead primary investigator James F. Howard, Jr, MD, Department of Neurology at The University of North Carolina School of Medicine, in a press release. “These results reinforce that C5 inhibition with predictable dosing is an important treatment option which provides sustained improvement of functional activities.”

Following the randomized control period of the CHAMPION-MG trial, 99.4% of participants (n=161) were administered ravulizumab-cwvz in the open label extension of the trial. At the time of data cut off, 113 patients reached 60 weeks with the efficacy analysis, including all patients administered ≥1 dose of ravulizumab-cwvz in the open label extension. Patients transitioned from placebo (n=83) showed rapid response at a similar magnitude and time course as those administered ravulizumab-cwvz during the randomized control period.

Credit: AstraZeneca

Safety and tolerability findings for ravulizumab-cwvz in the CHAMPION-MG trial were consistent with the known safety profile in the randomized control period of CHAMPION-MG and other approved indications. The most common adverse events occurring in more than or equal to 10% of patients in the randomized control period and/or open label extension were headache (16.6%) and diarrhea (13.6%).

Ravulizumab-cwvz was previously approved in the United States for the treatment of certain adults and children with paroxysmal nocturnal hemoglobinuriaand for certain adults and children with atypical hemolytic uremic syndrome.

“Alexion has pioneered the research of complement inhibition as a treatment approach for rare diseases, and we are continuing to innovate to benefit as many patients as possible,” said Gianluca Pirozzi, MD, PhD, senior vice president, head of Development and Safety, Alexion, in a press release. “These data are encouraging because they suggest Ultomiris has the potential to help a broader range of gMG patients, including those with milder symptoms, regain control of their lives and experience sustained clinical benefit through 60 weeks. We are deeply grateful for continued input and collaboration from the gMG community.”


Ultomiris demonstrated sustained improvements in functional activities and quality of life in adults with generalised myasthenia gravis through 60 weeks. AstraZeneca. News release. Accessed April 13, 2022.

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