In a phase 3 study of patients with atypical hemolytic uremic syndrome (aHUS), ravulizumab-cwvz (Ultomiris, Alexion) reached its primary objective.
aHUS is a chronic, progressive, debilitating, and extremely rare disease that affects children and adults. It can cause irreversible kidney damage and affect other vital organs, and can result in sudden and progressive kidney failure and premature death.
The primary endpoint of complete thrombotic microangiopathy (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 as meeting all three criteria simultaneously at least once, and those criteria had to be met for a minimum of 28 consecutive days.
A phase 3 trial of ravulizumab-cwvz in children and adolescents with aHUS is ongoing.
Ultomiris was approved by the FDA in December 2018 for adults with paroxysmal nocturnal hemoglobinuria (PNH).
Source: BioSpace, January 28, 2019