The U.S. Food and Drug Administration has accepted a supplemental Biologics License Application for Ultomiris (ravulizumab) submitted by Alexion, AstraZeneca Rare Disease, and granted the application Priority Review for use in adults with immunoglobulin A nephropathy (IgAN).
Priority Review is designated for drug applications that may offer significant improvements over existing therapies, whether through enhanced safety or efficacy, prevention of serious conditions, or improved patient adherence. With that designation in place, the FDA’s decision on the Ultomiris filing is expected in the fourth quarter of 2026 under the Prescription Drug User Fee Act timetable.
IgAN is described in the submission as a rare inflammatory kidney disease that can progress to chronic kidney disease and ultimately to end-stage kidney disease. The condition begins when the immune system produces abnormal immunoglobulin A (IgA) proteins that form immune complexes and deposit in the kidneys, producing damage. Those immune deposits trigger activation of the complement system, generating terminal complement-driven inflammation that injures glomeruli, the kidney’s filtering structures. The filing notes that more than 217,000 people in the United States have been diagnosed with IgAN.
"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 regulatory application is supported by a prespecified interim analysis from the I CAN Phase III trial, which AstraZeneca presented at the 2026 European Renal Association Congress. According to the interim data, Ultomiris produced a 46.6% reduction from baseline in 24-hour urine protein-to-creatinine ratio at week 34, compared with a 5.6% reduction for placebo, yielding a placebo-adjusted treatment effect of 43.4%.
Investigators observed an earlier onset of effect, with a 36.7% reduction at week 10 for patients receiving Ultomiris versus an 8.5% reduction for those on placebo, and this reduction in proteinuria persisted through week 34. The trial sponsor reported that the treatment effect was consistent across subgroups defined by demographic and baseline clinical characteristics.
The I CAN study’s primary endpoint is the change from baseline in estimated glomerular filtration rate (eGFR), which is scheduled to be measured at week 106. The submission therefore relies on the strength of the interim proteinuria results while the longer-term eGFR outcome remains to be assessed at the designated time point.
Safety observations in the interim analysis were said to align with Ultomiris’ previously characterized safety profile. Treatment was generally well tolerated in the trial and no new safety signals were identified in the interim dataset. If regulatory approval is granted, Ultomiris would become the first C5 complement inhibitor approved for this particular rare kidney disease.
Sectors affected: pharmaceutical and healthcare markets, particularly companies and investors focused on specialty medicines and rare disease therapeutics.