Ravulizumab significantly lowers the risk for relapse over placebo in patients with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4+ NMOSD), according to study results presented at the 2023 American Academy of Neurology (AAN) Annual Meeting, held from April 22 to 27, in Boston, Massachusetts.
In a global, open-label, phase 3 study (CHAMPION-NMOSD; ClinicalTrials.gov Identifier: NCT04201262), study authors recruited and assigned 105 participants with AQP4+ NMOSD to receive ravulizumab (n=58) or placebo (n=47). The primary endpoint was time to first adjudicated on-trial relapse and relapse risk reduction (RRR); secondary endpoints were adjudicated on-trial annualized relapse rate and clinically important worsening from baseline measured by the Hauser Ambulation Index (HAI) score.
No patients who received ravulizumab experienced adjudicated relapses after a median of 73.5 weeks of follow up (range, 11.0-117.7) compared with 20 adjudicated relapses after a median of 35 weeks of follow up (range, 1.9-117.7) in patients who received placebo (RRR, 98.6%; P <.0001). Fewer patients who received ravulizumab experienced clinically important HAI score worsening vs placebo (3.4% vs 23.4%; odds ratio, 0.16; 95% CI, 0.03-0.77).
Treatment with ravulizumab was associated with high rates of treatment-emergent adverse events (93.1%) and serious adverse events (13.8%). Of the 2 vaccinated patients who experienced meningococcal infection, 1 discontinued the trial although both recovered from the infection with no sequelae.
“Despite a longer follow-up (median treatment duration: 90.9 weeks), efficacy and safety remain consistent with the primary treatment period,” the study authors concluded, “No relapses were observed.”
Differences in baseline characteristics did not affect ravulizumab’s ability to lower risk for relapse and HAI score worsening in patients with AQP4+ NMOSD compared with placebo. No deaths were reported and no changes were made to ravulizumab’s known safety profile.
This article originally appeared on Neurology Advisor
Pittock S, Barnett M, Bennett J, et al. Efficacy and safety of ravulizumab in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder: outcomes from the phase 3 CHAMPION-NMOSD trial. Abstract presented at: 2023 AAN Annual Meeting; April 22-27, 2023; Boston, MA. Abstract S5.002.