April 28, 2022
AstraZeneca has announced FDA approval of #Ultomiris, a long-acting C5 complement inhibitor for the treatment of adults with generalized myasthenia gravis.
According to a company press release, Ultomiris (ravulizumab-cwvz) was approved for adult patients who are anti-acetylcholine receptor antibody-positive, which represents 80% of people with the disease. It is estimated that generalized myasthenia gravis affects approximately 90,000 people in the U.S.
Approval of the drug was based on positive results from the CHAMPION-MG phase 3 trial, in which ravulizumab-cwvz was more effective than placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile, a patient-reported assessment of the ability to perform daily tasks, total score at week 26.
“Despite recent advances, managing [generalized myasthenia gravis] is complex,” James F. Howard Jr., MD, professor of neurology at the University of North Carolina School of Medicine and lead primary investigator in the CHAMPION-MG trial, said in the release. “Earlier intervention can preserve function and quality of life. This approval offers patients, including those with milder symptoms, a long-acting C5 inhibitor with early onset and reliable efficacy.”
The safety profile of ravulizumab-cwvz was comparable to placebo in the trial and consistent with findings in phase 3 trials of the drug for paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. The most common adverse reactions were upper respiratory tract infection and diarrhea.