FDA approves drug for adults with generalized myasthenia gravis

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.

Results from the trial were recently published online in NEJM Evidence and presented at the 2022 American Academy of Neurology annual meeting.

“We are grateful for continued innovation and research into new treatment and dosing options to meet the needs of more patients and reduce the treatment burden,” Samantha Masterson, CEO of the Myasthenia Gravis Foundation of America, said in the release.