FDA approves Ultomiris for aHUS in adults and children

The US Food and Drug Administration 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.

Developed and marketed by US drugmaker Alexion Pharmaceuticals (Nasdaq: ALXN), 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 Dr Spero Cataland, Professor of Clinical Internal Medicine, Wexner Medical Center, The Ohio State University College of Medicine. “Clinical study results showed adult and pediatric patients had complete C5 inhibition following the first dose of Ultomiris. C5 inhibition was sustained over time with only six or seven infusions a year in adults - and that is important to consider for my patients,” he noted.