AstraZeneca Wins FDA Approval for Ultomiris in IgA Nephropathy

AstraZeneca has received accelerated approval from the US Food and Drug Administration (FDA) for Ultomiris (ravulizumab) to reduce protein levels in the urine of adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression.

Announced on October 21, 2026, the approval gives patients with this rare kidney disease access to a new treatment option. Ultomiris, developed by Alexion, AstraZeneca Rare Disease, is the first and only C5 complement inhibitor approved in the United States for this use.

IgA nephropathy is a kidney disease that develops when abnormal immune proteins build up in the kidneys, causing inflammation and damage. Over time, the disease can lead to chronic kidney disease and, in severe cases, kidney failure. One of its key signs is proteinuria, or high levels of protein in the urine, which can indicate kidney damage.

The FDA decision was supported by results from the Phase 3 I CAN clinical trial. In a planned interim analysis, Ultomiris reduced proteinuria by 43.4% compared with placebo after 34 weeks of treatment.

The study found that protein levels in the urine fell by 46.6% from baseline among patients receiving Ultomiris, compared with a 5.6% reduction among those receiving placebo. The improvement was observed as early as week 10 and continued through week 34.

The safety results were consistent with the medicine’s known safety profile, and no new safety concerns were identified in the interim analysis. The most common side effects reported among patients receiving Ultomiris included upper respiratory tract infections, nasopharyngitis and infusion-related reactions.

Ultomiris works by blocking C5, a protein involved in the complement system, which is part of the body’s immune defence. In IgA nephropathy, uncontrolled activation of this system can contribute to inflammation and damage to the kidneys. By blocking C5, the treatment aims to reduce this inflammatory activity and lower protein loss through the urine.

The medicine is given through an intravenous infusion following an initial loading dose, with maintenance treatment for adults administered every eight weeks.

However, the approval comes with an important limitation. Although Ultomiris reduced proteinuria in the clinical trial, it has not yet been established whether the treatment slows the long-term decline in kidney function in people with IgA nephropathy. The ongoing I CAN trial will assess changes in kidney function over 106 weeks, and these results are expected to help confirm the treatment’s clinical benefit. Continued approval may depend on verification of that benefit.

The FDA decision follows the agency’s earlier grant of Priority Review for the application in June 2026. Regulatory submissions based on the clinical results are also under review in Japan and other countries.

For AstraZeneca, the approval expands the use of Ultomiris beyond its existing indications for several rare diseases, including paroxysmal nocturnal haemoglobinuria, atypical haemolytic uraemic syndrome, generalised myasthenia gravis and neuromyelitis optica spectrum disorder.

The latest decision highlights continued efforts to develop targeted treatments for IgA nephropathy, a condition in which patients may remain at risk of kidney damage despite existing care. Further results from the I CAN trial will be important in determining whether Ultomiris can provide benefits beyond reducing proteinuria.