Ravulizumab Reduces Relapses in Patients with AQP4+ NMOSD

According to results of a study presented at the American Academy of Neurology (AAN) 2023 Annual Meeting, ravulizumab (Ultomiris;Alexion, Boston, MA) was effective at reducing relapses in patients with aquaporin-4 antibody positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD). Compared with those enlisted in the placebo arm, participants taking ravulizumab experienced no relapses after a median follow up of 73.5 weeks and experienced fewer declines in ambulation according to Hauser Ambulation Index (HA) scores. A significant percentage of those taking ravulizumab experienced treatment-emergent adverse events (93.1%).

CHAMPION-NMOSD (NCT04201262) is a global, external placebo-controlled, open-label multicenter phase 3 study designed to evaluate the efficacy and safety of ravulizumab in adult patients with AQP4+ NMOSD. The primary objective of the study was to determine the time to first relapse and relapse risk reduction (RRR). On day 1, 58 participants received a weight-based loading dose of ravulizumab through an IV infusion following by maintenance doses administered on day 15 and every 8 weeks for 2 years. The PREVENT (NCT01892345) placebo arm was used as the control arm.

Participants were included if they were AQP4+, diagnosed with NMOSD, had at least 1 attack or relapse in the last 12 months, an Expanded Disability Status Scale score ≤ 7, weighed at least 40 kg, and, if on contraceptives, were within the clinical guidelines. Participants were excluded if they had HIV or Neisseria meningitides infection, were treated with a complement inhibitor, or used rituximab, mitoxantrone, or immunoglobulin 3 weeks before the study.

The median range of follow up for those taking ravulizumab was 73.5 weeks during which time no relapses were reported in this group. There were 20 relapses reported in the control cohort (n=47) over a median follow up time of 36 weeks. The absolute risk reduction was 0.00 (0.04) with ravulizumab. Fewer patients had a worsening HAI score on ravulizumab (2/58; 3.4%) when compared to the control participants (11/47; 23.4%, 95% CI, 0.03–0.77)

NMOSD is a rare disease that affects the optic nerves and spinal cord as a relapsing-remitting illness. In the United States, around 15,000 people are diagnosed with this inflammatory disorder.