AstraZeneca’s rare-disease business, Alexion, has secured US Food and Drug Administration (FDA) accelerated approval for Ultomiris (ravulizumab) to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk of disease progression.
Announced on October 21, 2026, the approval follows positive results from a prespecified interim analysis of the Phase III I CAN trial. Ultomiris is the first and only C5 complement inhibitor approved in the US for reducing proteinuria in this patient population.
In the I CAN trial, Ultomiris achieved a 43.4% placebo-adjusted reduction in proteinuria at week 34. Patients receiving the treatment experienced a 46.6% reduction in 24-hour urine protein-to-creatinine ratio from baseline, compared with a 5.6% reduction among those receiving placebo.
The reduction was observed as early as week 10 and was sustained through week 34. The treatment’s safety profile was consistent with previous experience, with no new safety concerns identified in the interim analysis. Common adverse events included upper respiratory tract infections, nasopharyngitis and infusion-related reactions.
IgAN is a progressive kidney disease in which abnormal immunoglobulin A immune complexes accumulate in the kidneys, triggering inflammation and damage to the filtering structures. Persistent proteinuria is an important marker of kidney injury and is associated with an increased risk of progression to kidney failure.
Ultomiris works by inhibiting the C5 protein in the terminal complement pathway, which contributes to inflammation and kidney damage. Following an initial loading dose, adults receive the medicine intravenously every eight weeks, based on body weight.
The FDA granted accelerated approval based on the reduction in proteinuria. It has not yet been established whether Ultomiris slows the decline in kidney function in patients with IgAN.
The ongoing I CAN trial will assess changes in estimated glomerular filtration rate (eGFR) at week 106 to help confirm clinical benefit. The approval marks Ultomiris’ fifth indication, expanding its use beyond other complement-mediated rare diseases.
Written by: Pramit Hazra
Graphics by: Pramit Hazra
