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Ravulizumab effective in AQP4+ NMOSD

Presented By
Dr Sean Pittock, Mayo Clinic, USA
AAN 2023

In the open-label, phase 3 trial CHAMPION-NMOSD, ravulizumab significantly lowered the risk of relapse and worsening of the Hauser Ambulation Index (HAI) score versus placebo in participants with anti-aquaporin-4 antibody-positive neuromyelitis optica spectrum disorder (AQP4+ NMOSD).

Ravulizumab binds the same complement component 5 epitope as eculizumab. However, because of its longer half-life, it can be dosed more conveniently every 8 weeks instead of every 2 weeks. The safety and efficacy of ravulizumab were evaluated in the global, open-label phase 3 study CHAMPION-NMOSD (NCT04201262). The results were shared at the AAN 2023 meeting by lead author Dr Sean Pittock (Mayo Clinic, NY, USA) [1].

CHAMPION-NMOSD included 58 adult participants with anti-AQP4 NMOSD who had at least 1 attack or relapse in the 12 months before the screening visit. They were allowed to stay on stable supportive immunosuppressive therapy for the duration of the trial. All participants were given 2 meningococcal vaccines at least 2 weeks before starting ravulizumab treatment. The placebo arm of the PREVENT study (NCT01892345) served as an external comparator. The primary endpoints were time-to-first-on-trial relapse and relapse risk reduction (RRR). The median follow-up was 73.5 weeks for all 58 ravulizumab-treated participants and 36.0 weeks for 47 participants receiving a placebo in the PREVENT study.

In the ravulizumab group, no participants had a relapse, versus 20 participants in the control group (RRR 98.6%; P<0.0001). Also, significantly fewer participants in the ravulizumab group had clinically important worsening of HAI scores compared to placebo: 2/58 (3.4%) versus 11/47 (23.4%; P=0.023). In the ravulizumab group, 93.1% reported treatment-emergent adverse events (AEs). Serious AEs were seen in 13.8%, including 2 cases of meningococcal infection (2.4/100 patient-years), which recovered with no sequelae. Despite a longer follow-up period in the experimental group, efficacy and safety remained consistent with the primary treatment period. After a median of 91 weeks, still no relapses were observed.

    1. Pittock S. 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. Session S5.002, AAN 2023 Annual Meeting, 22–27 April, Boston, USA.

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