Alexion Pharma announces that the Phase 3 study of ULTOMIRIS met its primary objective in complement inhibitor-naïve patients with atypical hemolytic uremic syndrome (121.44)
In the initial 26 week treatment period, 53.6 percent of patients (95% CI [39.6%, 67.5%]) demonstrated complete thrombotic microangiopathy (TMA) response. ULTOMIRIS provided immediate and complete inhibition of the complement C5 protein that was sustained over the entire eight-week dosing interval. The primary endpoint of complete TMA response was defined by hematologic normalization and improved kidney function. The safety profile was consistent with that observed in two large Phase 3 studies in patients with paroxysmal nocturnal hemoglobinuria (PNH).
- Detailed results from this Phase 3 study will be presented at a future medical congress. A Phase 3 study of ULTOMIRIS in children and adolescents with aHUS is currently ongoing.