Phase III data augur label expansion for Alexion's Ultomiris

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) plans to submit new data to regulatory authorities that could expand the label of long-acting complement 5 (C5) inhibitor Ultomiris ravulizumab-cwvz to include atypical hemolytic uremic syndrome (aHUS).

Alexion said that Ultomiris met the primary endpoint in the Phase III ULTOMIRIS aHUS-311 trial in the indication. Among 56 complement inhibitor-naïve patients receiving the mAb in the open-label, international, single-arm trial, 53.6% had a complete thrombotic microangiopathy (TMA) response, as defined by hematologic normalization and improved kidney function, during the initial 26-week treatment period. A regulatory submission to FDA is planned for this half, with submissions in the EU and Japan to follow...