Subgroup Analyses of Ravulizumab Demonstrates Effectiveness Across Patient Populations: Michael Levy, MD, PhD

WATCH TIME: 5 minutes

"There were zero relapses, and it's very close to what we saw with eculizumab. My role was to then look at these pre-specified subgroups, like sex, age, geography, previous rituximab use, and determine if any of those subgroups were more or less likely to respond to ravulizumab. This was the plan all along but since there were zero relapses, it didn't make a lot of sense to have to divide zero by anything because it's just going to be zero."

Ravulizumab (Ultomiris; Alexion) was originally FDA-approved to treat adults with paroxysmal nocturnal hemoglobinuria in 2018 and later had its indication expanded to include children and adolescents in 2021. The regimen’s mechanism of action binds the same complement component 5 epitope as eculizumab (Soliris; Alexion) and its longer half-life enables an extended dosing interval, at 8 weeks versus 2 weeks. In a prespecified analysis of the CHAMPION-NMOSD (NCT04201262) open-label, phase 3 study, results showed ravulizumab was superior in aquaporin-4 (AQP4) antibody-positive neuromyelitis optica spectrum disorder (NMOSD) compared with placebo in prespecified subgroups by age, sex, race and geographic region.¹

These findings were presented as an abstract presentation at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27, in Boston, Massachusetts, by Michael Levy, MD, PhD. In CHAMPION-NMOSD, researchers evaluated the efficacy and safety of ravulizumab in 58 adult patients with anti-AQP4 NMOSD who had at least 1 attack or relapse in the 12 months prior to the screening visit. Adult patients received a weight-based intravenous loading dose of ravulizumab (2400–3000 mg), then a maintenance dose on day 15 (3000–3600 mg) and every 8 weeks following.

Levy, associate professor of neurology at Harvard Medical School, recently sat down with NeurologyLive® in an interview at the meeting to detail more about the subgroup analysis. Specifically, he talked about the effectiveness of ravulizumab as a treatment to prevent relapses in patients. He also spoke about the impact of subgroup analyses in clinical trials on furthering the research and expanding the profile of treatments such as ravulizumab for patients with NMOSD.

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REFERENCES
1. Levy M, Pittock S, Barnett M, et al. Efficacy subgroup analyses from the phase 3 CHAMPION-NMOSD trial in adults with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder. Presented at: 2023 AAN Annual Meeting; April 22-27, Boston, Massachusetts. Abstract 003.