AstraZeneca announced that its rare‑kidney‑disease drug Ultomiris met the primary endpoint of the Phase III I CAN trial, showing a statistically significant reduction in proteinuria in patients with immunoglobulin A nephropathy (IgAN). The interim analysis revealed that the drug lowered proteinuria as early as week 10 and continued to improve through week 34, a pattern that satisfies the trial’s co‑primary endpoint and supports a potential accelerated approval pathway in the United States and Europe.
Ultomiris is a C5 complement inhibitor that binds to the complement protein C5, preventing its cleavage into the pro‑inflammatory fragments C5a and C5b. By blocking terminal complement activation, the drug interrupts the inflammatory cascade that drives kidney damage in IgAN. The mechanism is particularly relevant because IgAN is characterized by abnormal IgA deposition that activates the complement system, leading to chronic inflammation and progressive kidney failure.
The IgAN market is estimated to affect more than 560,000 people worldwide, and the disease‑specific market is projected to grow from roughly $1.5 billion in 2025 to $11.6 billion by 2036, a CAGR of 18.6%. Ultomiris now competes with Novartis’s Fabhalta and other emerging candidates such as Atrasentan, Sibeprenlimab, and Zigakibart. If approved, the drug could capture a sizable share of this expanding market and provide a new high‑margin specialty therapy for AstraZeneca’s rare‑disease portfolio.
AstraZeneca’s rare‑disease strategy has been reinforced by the acquisition of Alexion Pharmaceuticals and recent deals with Amolyt Pharma and Gracell Biotechnologies. Ultomiris already generated $4.7 billion in worldwide sales in 2025, up 19% from 2024, and the IgAN indication would add a new revenue stream to the company’s portfolio of approved indications, which includes paroxysmal nocturnal hemoglobinuria, atypical hemolytic uremic syndrome, generalized myasthenia gravis, and neuromyelitis optica spectrum disorder.
"These positive data demonstrate that C5 complement inhibition with Ultomiris results in a rapid and clinically meaningful reduction in proteinuria as early as week 10 and underscores its potential as a disease‑modifying treatment option for IgAN," said Marc Dunoyer, CEO of Alexion, AstraZeneca Rare Disease. "Many people living with IgAN continue to progress to kidney failure, ultimately requiring dialysis or a transplant – outcomes that can place profound burden on patients' daily lives – despite advances in care. The interim I CAN results demonstrate that blocking terminal complement activation, a central driver of kidney inflammation in IgAN, with Ultomiris may play a promising role in reducing proteinuria," added Jonathan Barratt, MD, Mayer Professor of Renal Medicine, University of Leicester and I CAN trial investigator. He added, "We look forward to understanding the full clinical impact of Ultomiris in treating this disease following study completion at two years."
The trial will continue to week 106 to assess changes in estimated glomerular filtration rate (eGFR), a key marker of long‑term kidney function. AstraZeneca plans to submit data for accelerated approval in the United States and Europe, leveraging the interim proteinuria results as a surrogate endpoint while awaiting the final eGFR outcomes. The company’s next steps will involve regulatory filings, potential label expansion, and continued monitoring of safety and efficacy as the study progresses.
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