AstraZeneca's (AZN.US) ravulizumab injection has had a new indication proposed for priority review, according to the Center for Drug Evaluation (CDE) website of the National Medical Products Administration on October 12. The indication is for reducing proteinuria levels in adult patients with primary IgA nephropathy (IgAN).
Ravulizumab is a blockbuster product under AstraZeneca, with global sales of US$4.718 billion in 2025 (YOY +20.23%). Ravulizumab is a long-acting C5 complement inhibitor that provides rapid, sustained, and complete inhibition of complement C5. When the complement system is abnormally activated, its overreaction can cause the body to attack its own healthy cells. Ravulizumab works by targeting and inhibiting the activation of terminal complement C5 in the immune system.
Previously, ravulizumab has been approved and launched in multiple countries and regions worldwide, including Europe, the United States, Japan, and China. In China, the drug has been approved for three indications, including adult patients with anti-acetylcholine receptor (AChR) antibody-positive generalized myasthenia gravis (gMG), adult patients with neuromyelitis optica spectrum disorder (NMOSD), and adult and pediatric patients aged 1 month and older with paroxysmal nocturnal hemoglobinuria (PNH). The new indication proposed for priority review this time is IgA nephropathy.
In June 2026, AstraZeneca announced results from the Phase III I CAN study (ALXN1210-IgAN-320) of ravulizumab in treating adult IgA nephropathy. This was a global, Phase III, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of ravulizumab in adult patients with IgA nephropathy at risk of disease progression. The trial planned to enroll approximately 510 subjects from 28 countries across North America, South America, Europe, Asia, and Australia. The primary endpoints of the I CAN study were: change in proteinuria from baseline based on 24-hour urine protein-to-creatinine ratio (UPCR) (assessed at Week 34, conducted at the interim analysis), and change in estimated glomerular filtration rate (eGFR) from baseline (assessed at Week 106, conducted at the final analysis).
Interim analysis results showed that for the primary endpoint—change in proteinuria from baseline based on 24-hour UPCR—patients in the ravulizumab group experienced a clinically meaningful and significant reduction in proteinuria levels from baseline compared to the placebo group. The safety observed in this trial was consistent with the known safety profile of ravulizumab, with overall good tolerability and no new safety concerns identified. The most common adverse events (AEs) in the ravulizumab treatment group were upper respiratory tract infection, nasopharyngitis, and infusion-related reactions; the most common adverse events in the placebo group were upper respiratory tract infection, nasopharyngitis, and hypertension.
Dr. Jonathan Barratt, an investigator in the I CAN trial, stated: "For patients with IgA nephropathy, terminal complement activation is a key driver of inflammation and progressive loss of kidney function, which often leads to end-stage renal disease. These interim results demonstrate that ravulizumab, by targeting the terminal complement pathway, can rapidly and significantly reduce proteinuria, supporting its potential as a disease-modifying therapy for this rare and highly debilitating disease."
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