FDA grants accelerated approval to Ultomiris to reduce proteinuria in IgA nephropathy

The US Food and Drug Administration has granted accelerated approval to Ultomiris (ravulizumab) from Alexion, AstraZeneca Rare Disease, to reduce proteinuria (protein in the urine) in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. AstraZeneca announced the decision on 21 October 2026; the company calls it the first C5 complement inhibitor approved for this use.

The evidence

• The approval, which followed Priority Review, rests on a prespecified interim analysis of the randomised, placebo-controlled phase 3 I CAN trial.
• At week 34, the urine protein-to-creatinine ratio fell by 46.6% from baseline with Ultomiris versus 5.6% with placebo — a placebo-adjusted effect of 43.4%. The reduction was already visible at week 10.
• The most common side effects with Ultomiris were upper respiratory tract infection (11.3%), nasopharyngitis (9.2%) and infusion-related reactions (8.4%); AstraZeneca reports no new safety concerns.
• In adults the drug is given intravenously every eight weeks after a loading dose.

Limitations

This is an accelerated approval based on a surrogate marker. The label states that it has not been established whether Ultomiris slows kidney function decline in IgAN. Continued approval may depend on the ongoing I CAN trial, whose primary endpoint is the change in kidney filtration (eGFR) at week 106. The data cited here are interim and come largely from the company; fuller results are due at the American Society of Nephrology’s Kidney Week on 23 October. Fierce Pharma notes that Ultomiris enters a crowded market with drugs such as Tarpeyo, Filspari and Fabhalta. Patients should discuss treatment options with their nephrologist.