FDA approves Ultomiris to treat atypical hemolytic uremic syndrome.- Alexion Pharma

Alexion Pharmaceuticals, Inc. announced that the FDA 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.

Comment: Regulatory filings for marketing authorizations of Ultomiris for the treatment of aHUS in the European Union (EU) and Japan are under review with regulators..