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.
Among patients with evaluable data, ULTOMIRIS® allowed kidney function recovery (improvement by ≥1 eGFR category stage) vs baseline in 70% (30/43) of adults after 1 year§4
Among patients with aHUS who were on RRT at baseline, ULTOMIRIS® treatment led to dialysis discontinuation in 59% of paediatric patients (17/29) at Week 26, and 67% of adults (12/18) at Year 2‡4,5
§Adult patients receiving long-term dialysis (i.e. dialysis on a regular basis for ESRD) were excluded from the studies, and no conclusions can be drawn about renal efficacy of ULTOMIRIS® in these patients.1
‡Of the 27 patients who did not require dialysis at study entry, 19 patients remained off dialysis during the entire study period and 8 patients initiated dialysis during the study, with 2 of these patients discontinuing dialysis during the study. One of the patients that discontinued dialysis during the extension study period, then reinitiated dialysis and continued through study completion.3
‡Of the 27 patients who did not require dialysis at study entry, 19 patients remained off dialysis during the entire study period and 8 patients initiated dialysis during the study, with 2 of these patients discontinuing dialysis during the study. One of the patients that discontinued dialysis during the extension study period, then reinitiated dialysis and continued through study completion.3
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; TMA, thrombotic microangiopathy.