BOSTON -- (EUREKALERT!) -- Alexion Pharmaceuticals, Inc. today announced the first conference presentation of the topline Phase 3 data for ULTOMIRIS® (ravulizumab), the first and only long-acting C5 complement inhibitor administered every eight weeks, in adult patients with atypical hemolytic syndrome (aHUS). The previously announced data will be presented at the Annual Congress of the European Renal Association - European Dialysis and Transplant Association (ERA-EDTA), taking place June 13-16, 2019 in Budapest, Hungary:
Efficacy and safety of the long-acting complement C5 inhibitor ravulizumab in adult patients with atypical hemolytic uremic syndrome - Symposium 38, Regular Presentation, June 16, 2019, 09:30 a.m.1
Atypical HUS is a severe and chronic ultra-rare disease that affects both adults and children and can cause progressive and irreversible damage to vital organs, predominantly the kidneys, and lead to kidney failure (requiring dialysis or transplant) and premature death. The disease is characterized by inflammation and blood clotting in small blood vessels throughout the body, called thrombotic microangiopathy (TMA). This inflammation and blood clotting is mediated by chronic, uncontrolled activation of the complement system, which is part of the body's immune system.2,3,4,5,6
Early and careful diagnosis of aHUS is critical as many coexisting diseases and events are known or suspected to activate the complement cascade, and as patients may not necessarily present with the classic TMA triad of thrombocytopenia, hemolytic anemia and renal impairment,7 or may have less severe renal involvement.8 Available tests can help distinguish aHUS from other hemolytic diseases with similar symptoms such as HUS caused by Shiga toxin-producing Escherichia coli (STEC-HUS) and thrombotic thrombocytopenic purpura (TTP).6
Alexion submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for the approval of ULTOMIRIS in TMA, including aHUS, in April 2019 and plans to submit similar applications to the European Medicines Agency (EMA) and Pharmaceuticals and Medical Devices Agency (PMDA) in Japan later this year.