Alexion's Ultra-Rare Kidney Disease Drug Passes Phase III With Flying Colors

Boston-based Alexion’s Ultomiris (ravulizumab-cwvz) hit its primary objective in its Phase III study of complement inhibitor-naïve patients with the atypical hemolytic uremic syndrome (aHUS). aHUS is a chronic, progressive and debilitating ultra-rare disease. It affects children and adults and can lead to irreversible kidney damage as well as affecting other vital organs. It can result in sudden and progressive kidney failure and premature death. The disease symptoms include inflammation and the formation of blood clots in small blood vessels throughout the body mediated by uncontrolled activation of part of the immune system called the complement system. 
In the trial, 53.6 percent of patients receiving Ultomiris showed complete thrombotic microangiopathy response, giving immediate and complete inhibition of the complement C5 protein. This was sustained over the eight-week dosing interval. The primary endpoint of complete TMA response was defined by hematologic normalization and improved kidney function. Those receiving the drug had reduced thrombocytopenia, reduced destruction of red blood cells, and improved kidney function. TMA response was defined within the study as meeting all three criteria at the same time at least once and that criteria had to be hit for at least 28 consecutive days.