The FDA has approved eculizumab for the treatment of atypical hemolytic uremic syndrome (aHUS) in both children and adults, a potentially life-threatening condition.
The drug, marketed by Alexion Pharmaceuticals, Inc., of Cheshire, Conn., under the name Soliris, is the first and only FDA approved treatment for aHUS, an extremely rare genetic disease that can lead to renal failure, stroke, and myocardial infarction. The disease causes uncontrolled activation of the complement system, which results in the formation of blood clots in small blood vessels. More than half of patients with aHUS die, suffer permanent kidney damage, or require dialysis.
Eculizumab is a first-in-class terminal complement inhibitor that specifically targets uncontrolled complement activation. The new aHUS indication was granted under the FDA’s accelerated approval process based the findings of two prospective studies that included 37 adolescent and adult patients and a retrospective study of 19 pediatric patients.