AstraZeneca announced that the US Food and Drug Administration (FDA) has accepted and granted Priority Review to Alexion, AstraZeneca Rare Disease’s supplemental Biologics License Application (sBLA) for Ultomiris (ravulizumab) for the treatment of adults with immunoglobulin A nephropathy (IgAN), a rare and progressive kidney disease.

If approved, Ultomiris would become the first C5 complement inhibitor available for patients with IgAN, potentially offering a new treatment approach targeting complement-mediated inflammation associated with the disease.

The FDA grants Priority Review to therapies that may provide significant improvements over existing treatment options through enhanced efficacy, safety or by addressing serious medical conditions. The Prescription Drug User Fee Act (PDUFA) date, which marks the FDA’s anticipated regulatory decision, is expected in the fourth quarter of 2026.

The application is supported by results from a prespecified interim analysis of the Phase 3 CAN trial, recently presented at the 2026 European Renal Association (ERA) Congress. The study evaluated the efficacy and safety of Ultomiris in patients with IgAN.

Trial data showed that Ultomiris achieved a 46.6 percentage reduction in 24-hour Urine Protein Creatinine Ratio (UPCR) from baseline at week 34, compared with a 5.6 percentage reduction in the placebo group. This translated into a placebo-adjusted treatment effect of 43.4 percentage, indicating a significant reduction in proteinuria, a key marker of kidney damage.

The reduction in proteinuria was observed as early as week 10 and remained sustained through week 34. The treatment effect was consistent across various patient subgroups, including those with differing demographic characteristics, baseline clinical profiles and disease severity.

The study’s primary endpoint assessing changes in estimated glomerular filtration rate (eGFR), an important measure of kidney function, will be evaluated at week 106.

IgAN is a rare inflammatory kidney disease caused by abnormal immunoglobulin A (IgA) proteins that form immune complexes and deposit in the kidneys. These deposits activate the complement system, leading to terminal complement-driven inflammation that damages the glomeruli, the structures responsible for filtering blood. Over time, this damage can impair kidney function, resulting in chronic kidney disease and, in some cases, progression to end-stage kidney disease.

More than 217,000 people in the United States have been diagnosed with IgAN, highlighting the need for therapies that can slow disease progression and preserve kidney function.

Marc Dunoyer, Chief Executive Officer of Alexion, said that despite currently available treatments, many people living with IgAN continue to progress toward end-stage kidney disease. He noted that the Priority Review reflects the strength of the Phase III trial data and the potential of Ultomiris to address terminal complement-driven inflammation in the disease.

The safety profile observed in the I CAN trial was consistent with the known safety profile of Ultomiris. The treatment was generally well tolerated, and no new safety concerns were identified during the study.

Ultomiris is already approved for several complement-mediated disorders, and its potential expansion into IgAN represents another step in AstraZeneca and Alexion’s efforts to address rare diseases through complement science.