Ravulizumab: A Review in Atypical Haemolytic Uraemic Syndrome
Funding The preparation of this review was not supported by any external funding.
Authorship and Conflict of interest Yahiya Y. Syed is a salaried employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.
Ethics approval, Consent to participate, Consent to publish, Availability of data and material, Code availability Not applicable.
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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, Script and Slides
The video abstract, script and slide deck of images from the video, which have been peer reviewed and were uploaded to Figshare after the Ravulizumab in Atypical Haemolytic Uraemic Syndrome Adis Drug Evaluation was published, are sponsored by Alexion Pharmaceuticals, Inc.
The script and slide deck can be found here.
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