Ultomiris (ravulizumab) has been given Priority Review by the US FDA for immunoglobulin A nephropathy (IgAN) treatment. The supplemental Biologics License Application (sBLA) that was submitted by Alexion, AstraZeneca Rare Disease and accepted is based on results from a prespecified interim analysis of the I CAN phase 3 trial, which were recently presented at the 2026 European Renal Association (ERA) Congress.
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. More than 217,000 people are diagnosed with IgAN in the US.
Marc Dunoyer, Chief Executive Officer at 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. 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.”
In the I CAN phase 3 trial, Ultomiris demonstrated a 46.6% reduction in 24-hour urine protein creatinine ratio (UPCR) from baseline at week 34, compared to 5.6% in patients receiving placebo, resulting in a placebo-adjusted treatment effect of 43.4%.
The reduction in proteinuria was rapid, observed as early as week 10 with Ultomiris and sustained through 34 weeks, compared to placebo. These results were consistent across patient subgroups, reflecting diverse demographic and baseline clinical characteristics and disease severity. The trial’s primary endpoint of change from baseline in estimated glomerular filtration rate (eGFR) will be measured at week 106.
The safety profile observed in the I CAN trial was consistent with the known profile of Ultomiris and was generally well tolerated, with no new safety concerns identified.
The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for its regulatory decision, is anticipated during the fourth quarter of 2026.