The FDA Approves Ultomiris for Myasthenia Gravis

The FDA has approved Alexion/AstraZeneca’s Ultomiris (ravulizumab-cwvz) for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive, which represents 80% of people living with the disease.

This FDA action marks the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG. Generalized myasthenia gravis is a rare, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks in adult patients, following a loading dose.

Professor James F. Howard, Jr, M.D.

“Despite recent advances, managing gMG is complex,” Professor James F. Howard, Jr, M.D., department of neurology at The University of North Carolina School of Medicine and lead primary investigator, said in a press 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 approval was based on positive results from the CHAMPION-MG phase 3 trial, in which Ultomiris met the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile total score. Results were published recently online in NEJM Evidence and presented at the 2022 American Academy of Neurology Annual Meeting in April.

The safety profile was comparable to placebo and consistent with phase 3 trials of Ultomiris in paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome. The most common side effects were upper respiratory tract infection and diarrhea.