The US Food and Drug Administration (FDA) has granted accelerated approval to Ultomiris (ravulizumab) for the treatment of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN).
IgAN is characterized by the accumulation of IgA antibodies in the kidneys. In the early stages, patients with IgAN have no symptoms. The first sign of this condition may be blood in the urine. End-stage kidney disease may develop if the disease is not treated properly. In most instances, the cause of this condition is unknown; however, certain disorders have been linked with IgAN, such as cirrhosis of the liver, celiac disease, and HIV infection. The condition can also often be triggered by a viral illness.
Ravulizumab is a long-acting C5 complement inhibitor, providing immediate, complete and sustained complement inhibition. Following a loading dose, ravulizumab is administered intravenously every eight weeks in adults, or every four or eight weeks in paediatric patients (based on body weight).
The approval is based on positive results from a prespecified interim analysis of the I CAN trial. I CAN (NCT06291376) is a global, phase 3, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of ravulizumab in adults with IgAN who are at risk of disease progression.
Ravulizumab demonstrated a 46.6% reduction in 24-hour urine protein creatinine ratio (UPCR) from baseline at week 34, compared to 5.6% in patients receiving placebo, resulting in a placebo-adjusted treatment effect of 43.4%. The reduction in proteinuria was rapid, observed as early as week 10 with ravulizumab, and sustained through 34 weeks, compared to placebo.
The safety profile observed in the trial was consistent with the known profile of ravulizumab and was generally well tolerated, with no new safety concerns identified. Based on results from the prespecified interim analysis, the most common adverse events were upper respiratory tract infection, nasopharyngitis, and infusion-related reactions in those treated with ravulizumab and upper respiratory tract infection, nasopharyngitis, and hypertension in those receiving placebo.
This indication is approved under accelerated approval based on reduction of proteinuria. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the ongoing trial.
For more information, visit https://www.astrazeneca.com/media-centre/press-releases/2026/ultomiris-approved-in-us-in-igan.html
To learn more about IgAN and other rare kidney conditions, visit https://checkrare.com/diseases/kidney-and-urinary-diseases/