Ravulizumab: A Review in Atypical Haemolytic Uraemic Syndrome.

Abstract

Ravulizumab (Ultomiris®), a humanized monoclonal antibody that inhibits complement protein C5, is indicated for the treatment of atypical haemolytic uraemic syndrome (aHUS) in several countries, including the USA and those of the EU. Ravulizumab has been re-engineered from eculizumab to extend its terminal elimination half-life, resulting in a more convenient maintenance dosage regimen of once every 4–8 weeks compared with once every 2–3 weeks for eculizumab. In single-arm phase 3 trials, ravulizumab resolved thrombotic microangiopathy in 54% and 78% of treatment-naïve adult and paediatric patients with aHUS, respectively, within 26 weeks. Ravulizumab was also effective in patients with postpartum aHUS and paediatric patients who responded to eculizumab and later switched to ravulizumab. Ravulizumab was generally well tolerated, with no unexpected safety events. The most common treatment-related adverse events with ravulizumab in treatment-naïve patients include headache, diarrhoea and vomiting. With its convenient once every 4–8 weeks maintenance regimen, ravulizumab is an important treatment option for aHUS in adult and paediatric patients. Video abstract (MP4 80317 KB)Supplementary InformationThe online version contains supplementary material available at 10.1007/s40265-021-01481-6, which is available to authorized users.