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Ravulizumab effective in AQP4+ NMOSD

Presented by
Dr Sean Pittock, Mayo Clinic, NY, USA
AAN 2023
In the open-label, phase 3 trial CHAMPION-NMOSD, ravulizumab significantly lowered the risk of relapse and worsening on the Hauser Ambulation Index (HAI) compared with 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 relapsed 12 months before the screening visit. Participants were allowed to stay on stable supportive immunosuppressive therapy for the trial. All participants were given 2 meningococcal vaccines at least 2 weeks before starting the 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, compared with 20 participants in the control group (RRR 98.6%; P<0.0001). Also, significantly fewer participants in the ravulizumab group had clinically important worsening on the HAI compared with placebo: 2/58 (3.4%) versus 11/47 (23.4%; P=0.023).

In the ravulizumab group, 93.1% of participants 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 and no relapses were observed even after a median of 91 weeks.

  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. S5.002, AAN 2023 Annual Meeting, 22–27 April, Boston, USA.

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