Pharma giant AstraZeneca has been given a recommendation by the EU’s Committee for Medicinal Products (CHMP) for marketing authorisation for Ultomiris (ravulizumab), a treatment for adult patients with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive (Ab+).
Ultomiris is a C5 complement inhibitor that inhibits the C5 protein in the terminal complement cascade ‒ part of the body’s immune system. The complement cascade over-responds in NMOSD patients, which leads to the body to attack its own healthy cells.
The recommendation came after positive results from the CHAMPION-NMOSD phase 3 trial, which compared Ultomiris to an external placebo arm from the pivotal Soliris PREVENT clinical trial. 58 patients were enrolled, with participants having had a least one relapse in the 12 months prior to the study.
Data from the trial showed zero relapses were observed in Ultomiris patients with a median treatment duration of 73 weeks, and there were no new safety signals.
Orhan Aktas, MD, professor at the Department of Neurology, Medical Faculty at Heinrich-Heine-University, Düsseldorf, Germany, said: “Even one NMOSD relapse can lead to devastating long-term effects like vision loss, chronic pain and paralysis, which underscores the need for treatment innovations that help prevent relapses and optimise disease management. The sustained relapse risk reduction observed in the CHAMPION-NMOSD phase 3 trial supports the critical role this long-acting C5 complement inhibitor may have for the NMOSD community.”
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