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Ravulizumab in aquaporin-4-positive neuromyelitis optica spectrum disorder

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Item Type:Article
Title:Ravulizumab in aquaporin-4-positive neuromyelitis optica spectrum disorder
Creators Name:Pittock, S.J. and Barnett, M. and Bennett, J.L. and Berthele, A. and de Seze, J. and Levy, M. and Nakashima, I. and Oreja-Guevara, C. and Palace, J. and Paul, F. and Pozzilli, C. and Yountz, M. and Allen, K. and Mashhoon, Y. and Kim, H.J.
Abstract:OBJECTIVE: CHAMPION-NMOSD (NCT04201262) is a phase 3, open-label, externally controlled interventional study evaluating the efficacy and safety of the terminal complement inhibitor ravulizumab in adult patients with anti-aquaporin-4 antibody-positive (AQP4+) neuromyelitis optica spectrum disorder (NMOSD). Ravulizumab binds the same complement component 5 epitope as the approved therapeutic eculizumab but has a longer half-life, enabling an extended dosing interval (8 versus 2 weeks). METHODS: The availability of eculizumab precluded the use of a concurrent placebo control in CHAMPION-NMOSD; consequently, the placebo group of the eculizumab phase 3 trial PREVENT (n = 47) was used as an external comparator. Patients received weight-based intravenous ravulizumab on day 1 and maintenance doses on day 15, then once every 8 weeks. The primary endpoint was time to first adjudicated on-trial relapse. RESULTS: The primary endpoint was met; no patients taking ravulizumab (n = 58) had an adjudicated relapse (during 84.0 patient-years of treatment) versus 20 patients with adjudicated relapses in the placebo group of PREVENT (during 46.9 patient-years; relapse risk reduction, 98.6% [95% confidence interval, 89.7% to 100.0%], p < 0.0001). Median (range) study period follow-up time was 73.5 (11.0 to 117.7) weeks for ravulizumab. Most treatment-emergent adverse events were mild/moderate; no deaths were reported. Two patients taking ravulizumab experienced meningococcal infections. Both recovered with no sequelae; one continued ravulizumab treatment. INTERPRETATION: Ravulizumab significantly reduced relapse risk in patients with AQP4+ NMOSD, with a safety profile consistent with those of eculizumab and ravulizumab across all approved indications.
Keywords:Aquaporin 4, Complement Inactivating Agents, Neuromyelitis Optica, Recurrence
Source:Annals of Neurology
Page Range:1053-1068
Date:June 2023
Official Publication:https://doi.org/10.1002/ana.26626
PubMed:View item in PubMed

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