AstraZeneca (AZN.US) Relizumab's new indication is expected to be included in priority review

According to Jinse Finance APP, on October 12, the National Medical Products Administration Center for Drug Evaluation (CDE) website showed that a new indication for AstraZeneca (AZN.US)'s Ultomiris injection is proposed for priority review, applicable for reducing proteinuria levels in adult patients with primary IgA nephropathy (IgAN). Ultomiris is a blockbuster product under AstraZeneca, with global sales expected to reach $4.718 billion in 2025 (YOY +20.23%).

Ultomiris is a long-acting C5 complement inhibitor that provides rapid, sustained, and stable complete inhibition of complement C5. When the complement system is abnormally activated, its overreaction may cause the body to attack its own healthy cells. Ultomiris works by selectively inhibiting the activation of terminal complement C5 in the immune system.

Previously, Ultomiris has been approved for marketing in several countries and regions worldwide, including Europe, the United States, Japan, and China. Domestically, the drug has been approved for three indications: adult generalized myasthenia gravis (gMG) patients who are anti-acetylcholine receptor (AChR) antibody positive, adult neuromyelitis optica spectrum disorder (NMOSD) patients, and adult and children aged one month and above with paroxysmal nocturnal hemoglobinuria (PNH).

This proposed new indication for priority review is IgA nephropathy. In June 2026, AstraZeneca announced results of the phase III I CAN study (ALXN1210-IgAN-320) for Ultomiris in the treatment of adult patients with IgA nephropathy. This is a global, phase III, randomized, double-blind, placebo-controlled trial aimed at evaluating the efficacy and safety of Ultomiris in adult IgA nephropathy patients at risk of disease progression. The trial plans to recruit about 510 participants from 28 countries across North America, South America, Europe, Asia, and Australia.

The primary endpoints of the I CAN study are: change from baseline in proteinuria by 24-hour urine protein-to-creatinine ratio (UPCR) (assessed at week 34 during interim analysis), and change from baseline in estimated glomerular filtration rate (eGFR) (assessed at week 106 during the final analysis).

Interim analysis results showed that, for the primary endpoint—change from baseline in proteinuria based on 24-hour UPCR—patients in the Ultomiris group saw a significant and clinically meaningful reduction in proteinuria levels compared to baseline, relative to the placebo group.

The safety observed in this trial was consistent with the known safety profile of Ultomiris, with overall good tolerability and no new safety issues identified. The most common adverse events (AEs) in the Ultomiris treatment group were upper respiratory tract infection, nasopharyngitis, and infusion-related reactions; in the placebo group, the most common adverse events were upper respiratory tract infection, nasopharyngitis, and hypertension.

I CAN study investigator Dr. Jonathan Barratt stated: “For patients with IgA nephropathy, terminal complement activation is a key driver of inflammation and progressive loss of kidney function, often leading to end-stage renal disease. These interim results indicate that Ultomiris, by targeting the terminal complement pathway, can rapidly and significantly reduce proteinuria, supporting its potential as a disease-modifying therapy for this rare and life-threatening disease.”