Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced topline results from a Phase 3 study of weekly self-administered subcutaneous (SC) ULTOMIRIS® (ravulizumab-cwvz) in adults with paroxysmal nocturnal hemoglobinuria (PNH). The ongoing study met its primary objective of pharmacokinetic (PK)-based non-inferiority of ULTOMIRIS SC versus intravenous (IV) ULTOMIRIS at Day 71. Pending completion of the study, including collection of 12-month safety data as agreed to with the U.S. Food and Drug Administration (FDA), Alexion now expects to file for approval in the U.S. and E.U. for the ULTOMIRIS SC formulation and device combination in PNH and atypical hemolytic uremic syndrome (aHUS) in the third quarter of 2021.
“These data demonstrate that subcutaneous ULTOMIRIS may offer the same benefits of immediate, complete and sustained complement inhibition as the intravenous formulation, while also providing an additional treatment choice for those who would rather self-administer their medicine,” said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. “Delivered via a rapid, patient-friendly delivery device, subcutaneous ULTOMIRIS is an example of Alexion’s continued commitment to innovating for patients. It has the potential to be the first subcutaneous treatment option for both PNH and aHUS and may also offer improved quality of life for patients.”