Researchers reviewed 2-year data from two phase 3 studies on the efficacy of ravulizumab in pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS). Ravulizumab was found to improve blood health, kidney function, and quality of life while maintaining eGFR in patients with aHUS. This rare genetic disease is caused by unregulated complement system activation, leading to organ dysfunction. Ravulizumab, a complement C5 inhibitor, was effective in controlling the disease with fewer doses compared to eculizumab. Complete TMA response rates were high in both adult and pediatric patients, with quality of life improving and no meningococcal infections reported.
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Ravulizumab improves kidney function in patients with atypical hemolytic uremic syndrome
