Cambridge, United Kingdom – April 21, 2026

AstraZeneca announced that its complement inhibitor Ultomiris® (ravulizumab) achieved statistically significant and clinically meaningful reduction in proteinuria in adults with immunoglobulin A nephropathy (IgAN) in the Phase III I CAN clinical trial, marking a major advancement in the treatment of this rare and progressive kidney disease. The positive interim analysis demonstrates rapid therapeutic impact as early as week 10, reinforcing the drug’s potential as a disease-modifying therapy targeting complement-driven inflammation.

Phase III Trial Demonstrates Significant Reduction in Proteinuria

The I CAN Phase III trial, a global randomized, double-blind, placebo-controlled study, met its primary endpoint by showing a significant reduction in proteinuria, measured by the 24-hour urine protein creatinine ratio (UPCR) at week 34. This reduction is clinically meaningful, as proteinuria is a key indicator of kidney damage and disease progression in IgAN patients.

Notably, the therapy demonstrated rapid onset of action, with measurable reductions in proteinuria observed as early as week 10, highlighting its potential to slow disease progression early in the treatment course. The trial continues to evaluate long-term outcomes, including changes in estimated glomerular filtration rate (eGFR) at week 106, which will further determine the therapy’s impact on kidney function preservation.

The safety profile observed in the study was consistent with previously established data, with no new safety concerns identified, supporting the therapy’s favorable benefit-risk profile.

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Targeting Complement Pathway to Address Disease Mechanism

IgAN is a chronic inflammatory kidney disease caused by abnormal deposition of IgA immune complexes in the kidneys, leading to activation of the complement system and progressive renal damage. Ultomiris works by inhibiting the C5 protein in the terminal complement cascade, thereby preventing excessive immune activation and reducing inflammation-driven kidney injury.

This mechanism positions Ultomiris as a potential disease-modifying therapy, rather than a treatment that only manages symptoms. By targeting the underlying biological drivers of IgAN, the therapy offers a promising approach to reducing long-term complications such as chronic kidney disease and end-stage kidney failure.

Given that more than 560,000 people across major global markets are affected by IgAN, and a significant proportion are at risk of disease progression, the need for effective targeted therapies remains high.

Regulatory Pathway and Future Clinical Impact

Following these positive interim results, AstraZeneca plans to engage with regulatory authorities and pursue accelerated approval pathways in key markets, reflecting the therapy’s potential to address a serious condition with significant unmet medical need.

Ultomiris is already approved for multiple rare diseases, including paroxysmal nocturnal haemoglobinuria (PNH), atypical haemolytic uraemic syndrome (aHUS), and generalized myasthenia gravis, demonstrating its established clinical utility and safety profile. The expansion into IgAN further strengthens its role as a leading complement inhibitor in rare disease therapeutics.

The continued development of Ultomiris underscores the growing importance of precision immunology and complement-targeted therapies in addressing complex inflammatory diseases. As additional data from the I CAN trial emerges, the therapy has the potential to transform the standard of care for IgAN, offering patients a new option to slow disease progression and improve long-term outcomes.

Source: AstraZeneca press release