AstraZeneca’s (LSE:AZN) rare disease division, Alexion, has received a regulatory boost after the U.S. Food and Drug Administration accepted and granted priority review to a supplemental biologics licence application for Ultomiris (ravulizumab) as a treatment for adults with immunoglobulin A nephropathy (IgAN).
Priority review status is reserved for therapies that have the potential to provide meaningful improvements over existing treatment options, whether through enhanced efficacy, improved safety or other significant clinical benefits. The FDA is expected to make its decision during the fourth quarter of 2026.
Targeting a Serious Rare Kidney Disorder
Immunoglobulin A nephropathy is a chronic inflammatory kidney disease that can progressively impair kidney function and may ultimately lead to end-stage kidney disease. More than 217,000 people in the United States are diagnosed with the condition.
The disease occurs when immunoglobulin A deposits accumulate in the kidneys, triggering inflammation and potentially causing long-term damage to the organs’ filtering capability.
Phase III Trial Demonstrated Significant Reduction in Proteinuria
The regulatory submission is supported by interim data from the Phase III I CAN study evaluating Ultomiris in patients with IgAN.
Results showed that patients receiving Ultomiris achieved a 46.6% reduction in 24-hour urine protein-creatinine ratio from baseline at week 34, compared with a 5.6% reduction among patients receiving placebo. This translated into a placebo-adjusted treatment effect of 43.4%.
According to the study findings, reductions in proteinuria were evident as early as week 10 and were maintained throughout the 34-week assessment period.
Kidney Function Endpoint Still Ongoing
While the interim analysis focused on proteinuria reduction, the trial’s primary endpoint remains the change in estimated glomerular filtration rate (eGFR), a key measure of kidney function.
This endpoint will be assessed at week 106, providing additional evidence on the long-term impact of Ultomiris on disease progression and kidney health.
Safety Profile Remains Consistent
AstraZeneca reported that the safety findings from the I CAN trial were consistent with the established safety profile of Ultomiris.
The treatment was generally well tolerated, and the company said no new safety concerns were identified during the study.
More About AstraZeneca
AstraZeneca is a global biopharmaceutical company focused on the discovery, development and commercialisation of medicines across oncology, rare diseases, cardiovascular, renal and metabolic disorders, respiratory diseases and immunology.
Through its Alexion division, the company specialises in treatments for rare and serious diseases, with Ultomiris forming part of a portfolio of therapies designed to address conditions driven by complement system dysregulation.
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