The approval by the Food and Drug Administration (FDA) was based on positive results from the CHAMPION-MG Phase III trial, in which Ultomiris was superior to placebo in the primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26, a patient-reported scale that assesses patients’ abilities to perform daily activities.
This FDA action marks the first and only approval for a long-acting C5 complement inhibitor for the treatment of gMG, a rare, debilitating, chronic, autoimmune neuromuscular disease that leads to a loss of muscle function and severe weakness. The diagnosed prevalence of gMG in the US is estimated at approximately 90,000.7
James F. Howard, Jr, MD, professor in the UNC Department of Neurology, and lead primary investigator in the CHAMPION-MG trial said: “Despite recent advances, managing gMG is complex. 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.”
Read the full press release from AstraZeneca, which released news of the FDA approval.
James F. Howard, Jr, MD