FDA accepts sBLA for Ultomiris to treat atypical hemolytic uremic syndrome (aHUS)

Alexion Pharmaceuticals, Inc. announced that the FDA has accepted for priority review the company’s supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab-cwvz), the company’s long-acting C5 complement inhibitor, for the treatment of people with atypical hemolytic uremic syndrome (aHUS) in order to inhibit complement-mediated thrombotic microangiopathy (TMA). The FDA has set a target action date of 19 October 2019 under the Prescription Drug User Fee Act (PDUFA).

The sBLA is based on previously announced results, which were recently presented at the European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) Congress , from the Phase III study of Ultomiris in people with aHUS, which met the primary endpoint of complete TMA response, defined by hematologic normalization and improved kidney function.