Efficacy of ULTOMIRIS®

The efficacy of Ultomiris® was evaluated in two pivotal phase 3 trials, with a primary endpoint of complete TMA response.1,2 Visit the Study design page to get more information on the methodology of the clinical trials
Among patients with evaluable data, ULTOMIRIS® allowed kidney function recovery (improvement by ≥1 eGFR category stage) vs baseline in 100% (16/16) of paediatric and adolescent patients after 1 year§,1
Among patients with aHUS who were on RRT at baseline, ULTOMIRIS®
treatment led to dialysis discontinuation in 83% of paediatric patients (5/6) at Week 26, and 100% of paediatric patients (6/6) at Year 21,4
§Paediatric patients receiving long-term dialysis (i.e. dialysis on a regular basis for ESRD) were excluded from the study, and no conclusions can be drawn about renal efficacy of ULTOMIRIS® in these patients.1
Adverse Event Reporting

Please report any adverse events via your national reporting system. Adverse events can also be reported to Alexion Pharmaceuticals by contacting: https://contactazmedical.astrazeneca.com/
C5, complement component 5; aHUS, atypical haemolytic uraemic syndrome; eGFR, estimated glomerular filtration rate; ESRD, end-stage renal disease; RRT, renal replacement therapy; TMA, thrombotic microangiopathy.
Ariceta G, et al. Kidney Int. 2021;100(1):225–237. Rondeau E, et al. Kidney Int. 2020;97(6):1287–1296 ULTOMIRIS® EU Summary of Product Characteristics available at https://www.ema.europa.eu/en/documents/product-information/ultomiris-epar-product-information_en.pdf Last accessed: December 2024. Dixon BP, et al. Kidney Med. 2024;6(8):100855;