Business
AstraZeneca : Ultomiris granted Priority Review in the US as treatment for adults with immunoglobulin A nephropathy
AstraZeneca : Ultomiris granted Priority Review in the US as treatment for adults with immunoglobulin A
About this update from Astrazeneca Plc
Based on I CAN Phase III trial results from prespecified interim analysis in which Ultomiris demonstrated 43.4% reduction in proteinuria vs placebo at 34 weeksIf approved, Ultomiris would be the first C5 complement inhibitor available for this rare kidney disease Alexion, AstraZeneca Rare Disease's supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab) has been accepted and granted Priority Review by the US Food and Drug Administration (FDA) for the treatment of adults with immunoglobulin A nephropathy (IgAN). The FDA grants Priority Review to applications for medicines that, if approved, would offer significant improvements over available treatment options by demonstrating safety or efficacy improvements, preventing serious conditions or enhancing patient compliance. The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for its regulatory decision, is anticipated during the fourth quarter of 2026. IgAN is a rare, inflammatory disease of the kidneys that can lead to chronic kidney disease and progress to end-stage kidney disease. It begins when the body develops abnormal IgA proteins resulting in immune complexes that are deposited in the kidneys causing damage. The deposition of these complexes activates the complement system, leading to terminal complement-driven inflammation. This results in damage and loss of essential parts of the kidney, including cells in the glomeruli, the part of the kidneys that filters and cleans the blood. Over time, this damage impacts the ability of the kidneys to function properly.1 More than 217,000 people are diagnosed with IgAN in the US.2 Marc Dunoyer, Chief Executive Officer, Alexion, said: "Despite available treatments, people living with IgAN often progress to end-stage kidney disease, underscoring the urgent need for new disease-modifying approaches. Building on our pioneering leadership in complement science, this Priority Review reflects the strength of the interim analysis data from the I CAN trial and the potential of Ultomiris as the first C5 complement inhibitor to address terminal complement-driven inflammation in IgAN." The sBLA is based on results from a prespecified interim analysis of the I CAN Phase III trial, which were recently presented at the 2026 European Renal Association (ERA) Congress.3 In the trial, Ultomiris demonstrated a 46.6% reduction in 24-hour urine p...