AstraZeneca announced that Ultomiris (ravulizumab-cwvz), an Alexion, AstraZeneca Rare Disease drug, has been approved by the FDA as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The approval was based on positive results from the CHAMPION-NMOSD phase 3 trial. In the trial, Ultomiris was compared to an external placebo arm from the pivotal Soliris PREVENT clinical trial. Ultomiris met the primary endpoint of time to first on-trial relapse as confirmed by an independent adjudication committee. Zero relapses were observed among Ultomiris patients with a median treatment duration of 73 weeks. Overall, the safety and tolerability of Ultomiris in the CHAMPION-NMOSD trial were consistent with previous clinical studies and real-world use, and no new safety signals were observed. 

“With the FDA approval, patients now have the option of a long-acting C5 inhibitor treatment that showed zero relapses in the pivotal CHAMPION-NMOSD trial, supporting the primary goal of relapse prevention in treating NMOSD,” Sean J. Pittock, MD, lead primary investigator in the CHAMPION-NMOSD trial, said in a news release. Dr. Pittock is director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo’s Neuroimmunology Laboratory. Alexion, AstraZeneca Rare Disease is a group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc.