Alexion Pharmaceuticals reports that the FDA has approved ULTOMIRIS® (ravulizumab-cwvz) for the treatment of atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) for adult and pediatric (one month of age and older) patients. This is the first pediatric approval for ULTOMIRIS.
Atypical HUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Atypical HUS can cause sudden organ failure or a slow loss of function over time—potentially resulting in the need for a transplant, and in some cases, death.
“The primary approach to treatment is to prevent the body from attacking itself, through the inhibition of uncontrolled complement activation, referred to as C5 inhibition,” said Spero Cataland, M.D., Professor of Clinical Internal Medicine, Wexner Medical Center, The Ohio State University College of Medicine. more