AstraZeneca plans to seek accelerated approval of Ultomirisin kidney disease after the drug showed benefit after just 34 weeks, meeting its first primary endpoint.
In the Phase III I CAN study (NCT06291376), patients with immunoglobulin A nephropathy (IgAN) treated with Ultomiris (ravulizumab) saw a statistically significant and clinically meaningful reduction of proteinuria, based on 24-hour urine protein creatinine ratio (UPCR), after week 34.
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The co-primary endpoint of change from baseline in estimated glomerular filtration rate (eGFR) will be measured at week 106.
Data from secondary endpoints including change in albuminuria, partial remission and change in Functional Assessment of Chronic Illness Therapy (FACIT) are not yet available.
Jonathan Barratt, MD, Mayer Professor of Renal Medicine, University of Leicester, UK, and I CAN trial investigator, said: “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. We look forward to understanding the full clinical impact of Ultomiris in treating this disease following study completion at two years.”
The ongoing study, being conducted by AstraZeneca’s rare disease company Alexion, is set to enrol 510 patients with IgAN. IgAN is a rare, inflammatory disease of the kidneys that can lead to chronic kidney disease (CKD) and progress to end-stage kidney disease (ESKD). AstraZeneca reports that more than 560,000 people are diagnosed with IgAN in the US, EU5 and Japan.
The safety profile observed in this trial was consistent with the known profile of Ultomiris, with no new safety concerns identified.
Based on the early data, Alexion will seek accelerated approval for the monoclonal antibody (mAb) in key markets and will present these results at a forthcoming medical meeting.
Other companies are making their mark on IgAN with Vertex seeking approval of povetacicept after it met the primary endpoint in a Phase III trial. Patients treated received a 52% reduction in UPCR after 36 weeks.
Novartis has also had recent success in IgAN in the Phase III ALIGN study, which found that Vanrafia (atrasentan) slowed kidney function decline. Patients saw a 2.39ml / min / 1.73m² difference in eGFR compared to placebo at week 136, four weeks after ending treatment. The drug gained accelerated approval in April 2025.
The market has also heated up in recent years, with Otsuka’s Voyxact (sibeprenlimab-szsi), having gained US Food and Drug Administration (FDA) approval in November 2025. More established therapies include Calliditas Therapeutics’ Tarpeyo (budesonide), which gained accelerated approval from the FDA in 2021, and Travere Therapeutics’ Filspari (sparsentan), which gained accelerated approval in 2023.
