Alexion ULTOMIRIS receives FDA approval for Atypical Hemolytic Uremic Syndrome
Alexion Pharmaceuticals announced that the U.S. Food and Drug Administration approved ULTOMIRIS for the treatment of atypical hemolytic uremic syndrome to inhibit complement-mediated thrombotic microangiopathy for adult and pediatric 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. The FDA approval is based on data from two global, single-arm open-label studies of ULTOMIRIS - one in adults and one in children, referred to as pediatrics in the study - with aHUS. The pediatric study is ongoing and a total of 14 out of 16 children were enrolled and included in the interim analysis. Efficacy evaluation of Complete TMA Response was defined by hematologic normalization parameters and improved kidney function. In the initial 26-week treatment periods, 54% of adults and 71% of children demonstrated Complete TMA Response. Treatment with ULTOMIRIS resulted in reduced thrombocytopenia in 84% of adults and 93% of children, reduced hemolysis in 77% of adults and 86% of children, and improved kidney function in 59% of adults and 79% of children. The most frequently observed adverse reactions reported in these studies were upper respiratory tract infection, diarrhea, nausea, vomiting, headache, hypertension and pyrexia. Serious meningococcal infections have occurred in patients treated with ULTOMIRIS. To minimize the risk for patients, specific risk-mitigation plans, including a REMS, have been established for ULTOMIRIS. Regulatory filings for marketing authorizations of ULTOMIRIS for the treatment of aHUS in the European Union and Japan are under review with regulators.