FDA approves AstraZeneca’s Ultomiris for rare nerve disease NMOSD

ULTOMIRIS or ravulizumab-cwvz, has been approved in the United States 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 by the US Food and Drug Administration FDA was based on positive results from the CHAMPION-NMOSD Phase III trial, which were published in the Annals of Neurology. 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 and recurrent and may result in permanent disability.6-8 The diagnosed prevalence of adults with NMOSD in the US is estimated at approximately 6,000.9-11…Sean J. Pittock, MD, Director of Mayo Clinic’s Center for Multiple Sclerosis and Autoimmune Neurology and of Mayo’s Neuroimmunology Laboratory and lead primary investigator in the CHAMPION-NMOSD trial, said: “C5 inhibition has been proven to offer efficacy in reducing the risk of NMOSD relapses by blocking the complement system, a part of the immune system, from attacking healthy cells in the spinal cord, optic nerve and brain. With today’s 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.”