Ultomiris (ravulizumab-cwvz) has been granted approval by the US Food and Drug Administration (FDA) as the premier long-acting C5 complement inhibitor for treating adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD). The endorsement was based on favorable outcomes from the CHAMPION-NMOSD Phase III trial, showcased in the Annals of Neurology. Ultomiris, compared against an external placebo arm from the Soliris PREVENT clinical trial, achieved its primary objective of delaying the first on-trial relapse, with no relapses observed among Ultomiris patients during a median treatment duration of 73 weeks.
NMOSD, a rare autoimmune condition affecting the central nervous system, presents debilitating symptoms, often marked by unpredictable relapses leading to severe neurological complications. Dr. Sean J. Pittock, the lead primary investigator in the CHAMPION-NMOSD trial, emphasized the efficacy of C5 inhibition in curbing relapse risks by obstructing the complement system’s attack on healthy CNS cells. This approval provides patients with a long-acting treatment option, pivotal in preventing NMOSD relapses.
Marc Dunoyer, CEO of Alexion, expressed pride in delivering a transformative treatment option with Ultomiris, capable of eliminating relapses through a convenient dosing regimen every eight weeks. Safety profiles of Ultomiris in the CHAMPION-NMOSD trial remained consistent with prior studies, with no new safety concerns identified. The most common adverse events included COVID-19, headache, back pain, arthralgia, and urinary tract infections.
Ultomiris, with its unique mechanism inhibiting the C5 protein, provides immediate, complete, and sustained complement inhibition, administered intravenously every eight weeks following a loading dose. Besides NMOSD, Ultomiris has garnered approvals for various other conditions like generalized myasthenia gravis (gMG), paroxysmal nocturnal hemoglobinuria (PNH), and atypical hemolytic uremic syndrome (aHUS), showcasing its versatility in treating hematologic and neurologic disorders.
Ultomiris emerges as a groundbreaking therapeutic option, not only for NMOSD but also for a spectrum of hematologic and neurologic indications, promising a future with reduced relapse risks and improved quality of life for patients.