Ravulizumab (Ultomiris®) has the potential to cause rapid and sustained improvements in functional and clinical measures for patients with generalized myasthenia gravis (MG) who are seropositive for anti-acetylcholine receptor (anti-AChR) antibodies, according to the results of an open-label extension (OLE) study published in the Journal of Neurology.

For patients who received ravulizumab during the randomized controlled period (RCP) of the study, improvements in scores continued and were maintained up to the 60-week endpoint for the Myasthenia Gravis-Activities of Daily Living (MG-ADL), revised 15-item Myasthenia Gravis Quality of Life (MG-QOL15r), Neurological Quality of Life (Neuro-QoL) Fatigue subscale, and Quantitative Myasthenia Gravis (QMG) scores.

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Improvements of 4 to 5 points were observed in the MG-ADL, QMG, and MG-QOL15r from baseline until week 60 of treatment (all P <.0001). The Neuro-QoL Fatigue score improved by 10.2 points (P <.0001) during the same time period.

Patients who had received placebo during the RCP of the trial saw rapid improvements in scores by week 2 or 4 of the OLE (all P <.0001). These scores continued and resulted in improvements of 3.3 points for MG-ADL, 3.8 points for QMG, 5.4 points for MG-QOL15r, and 14.0 points for Neuro-QoL Fatigue from RCP baseline to week 60.

Ravulizumab treatment also led to a reduction in clinical deterioration event rates. The event rates per 100 patient-years were 44.4 in the 1-year period before the start of the study and 61.6 in patients who received placebo during the RCP. The rate was only 17.8 for patients receiving ravulizumab in the RCP and OLE portions of the study, corresponding to a 59.8% reduction for ravulizumab compared to the prestudy values. There was a 71.1% reduction when comparing ravulizumab and the placebo.

The study initially enrolled 175 patients in the RCP from 85 centers in 13 countries, with 162 patients completing the RCP. Of those, 161 continued onto the OLE, with 11 patients withdrawing before week 60. The set of patients who completed the OLE included 55 patients who received ravulizumab during both phases of the study and 58 who initially received placebo during the RCP.


Meisel A, Annane D, Vu T, et al; CHAMPION MG Study Group. Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension. J Neurol. Published online April 27, 2023. doi:10.1007/s00415-023-11699-x