Ravulizumab (ULTOMIRIS®) is the first long-acting complement C5 inhibitor (administered intravenously every 8 weeks) to be approved in several countries globally, for adults with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor antibody-positive (AChR Ab+). In the phase III CHAMPION MG trial, intravenous ravulizumab was associated with statistically significant improvements in the MG-Activities of Daily Living scale at week 26 of treatment compared with placebo in adults with AChR Ab+ gMG. Improvements in the Quantitative MG scale total score were also statistically significantly higher in ravulizumab than placebo recipients. These improvements were sustained to week 26 of treatment. Ravulizumab was generally well tolerated; the most common treatment-emergent adverse events were headache, diarrhoea and nausea. Efficacy and tolerability data for up to 1 year from the ongoing open-label extension phase are consistent with those from the randomized, placebo-controlled phase; further results are awaited with interest. Thus, ravulizumab is an efficacious, generally well tolerated and convenient treatment option in adults with AChR Ab+ gMG, expanding the options available for gMG management.
Generalised myasthenia gravis (gMG) is a rare chronic condition that affects the muscles, making them become abnormally tired and weak after use. Prevalence can vary (5.3–35 per 100,000 people) and is steadily rising. Management of gMG can involve modifying or suppressing the immune system, symptom management and/or surgical removal of the thymus gland. Complement C5 inhibitors are another treatment option for patients with gMG. Ravulizumab (ULTOMIRIS®) is the first long-acting complement C5 inhibitor (administered intravenously every 8 weeks) to be approved in several countries globally for the treatment of adults with gMG who are anti-acetylcholine receptor antibody-positive (AChR Ab+). Ravulizumab is associated with long-lasting improvements in activities of daily living and disease status in adults with AChR Ab+ gMG, as demonstrated in a phase III clinical trial. In this trial, ravulizumab was generally well tolerated; headache, diarrhoea and nausea were the most common adverse events. Although there is a potential risk for adverse reactions with ravulizumab treatment, including serious meningococcal infections, other infections and infusion-related reactions, no meningococcal infections occurred and the incidence of infusion-related reactions was low in patients with gMG. The efficacy and tolerability of ravulizumab were sustained for up to 1 year of treatment; further results are awaited with interest. Thus, ravulizumab is an efficacious, generally well tolerated and convenient treatment option in adults with AChR Ab+ gMG, expanding the options available for gMG management.
© 2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.