The FDA has granted accelerated approval to AstraZeneca’s Ultomiris to reduce protein in the urine of adults with primary immunoglobulin A nephropathy who risk disease progression.
The decision makes Ultomiris, or ravulizumab, the first C5 complement inhibitor approved for this use. The drug is administered intravenously every eight weeks after a loading dose, with dosing based on body weight.
Approval rests on reduced proteinuria, rather than demonstrated preservation of kidney function. It has not been established whether Ultomiris slows kidney function decline in these patients.
Continued approval may depend on confirmation of clinical benefit in the ongoing Phase 3 I CAN trial. The study will assess changes in estimated glomerular filtration rate at Week 106, with results intended to support traditional approval.
In a prespecified interim analysis, Ultomiris reduced the 24-hour urine protein-to-creatinine ratio by 46.6% from baseline at Week 34, compared with 5.6% for placebo. The placebo-adjusted treatment effect was 43.4%, with a p-value below 0.0001.
Reductions were observed by Week 10 and persisted through Week 34.
IgA nephropathy develops when abnormal immunoglobulin A proteins form immune complexes that accumulate in the kidneys, triggering inflammation and damage. The disease can progress to kidney failure.
The trial identified no new safety concerns. Common adverse events among Ultomiris recipients included upper respiratory tract infection, affecting 11.3%; nasopharyngitis, 9.2%; and infusion-related reactions, 8.4%.
Late-breaking trial results will be presented at the American Society of Nephrology’s Kidney Week. Regulatory submissions based on the interim findings are under review in Japan and additional countries.
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