The FDA Grants Accelerated Approval of ULTOMIRIS (ravulizumab-cwvz) for Adults with IgA Nephropathy 

October 2026 — NephCure celebrates another landmark milestone for the rare kidney disease (RKD) community following the U.S. Food and Drug Administration’s (FDA) accelerated approval of ULTOMIRIS® (ravulizumab-cwvz) as the first and only C5 complement inhibitor to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) who are at risk for disease progression. 

The FDA’s accelerated approval program provides patients with earlier access to innovative treatments for serious conditions with high unmet medical needs. This pathway relies on a surrogate endpoint, in this case, the reduction of proteinuria (excess protein in the urine), which serves as a key predictor of long-term kidney protection. The ongoing Phase III I CAN trial will continue evaluating estimated glomerular filtration rate (eGFR) outcomes to support traditional approval. 

IgAN is a rare kidney disease in which Immunoglobulin A (IgA) builds up in the kidney. IgA is a protein in the blood and is also part of the immune system. Excess IgA can cause inflammation in the kidney and over time, this leads to scarring in the kidney tissue. The severity of kidney disease caused by IgAN varies from person to person.  

Developed by Alexion, AstraZeneca Rare Disease, ULTOMIRIS is a targeted C5 complement inhibitor designed to deliver sustained terminal complement inhibition. By blocking this central driver of complement-mediated kidney inflammation, ULTOMIRIS directly addresses the underlying inflammatory cascades that accelerate tissue damage and protein loss in IgAN. 

The FDA approval is based on robust data from a prespecified interim analysis of the Phase III I CAN clinical trial, which demonstrated: 

• A 43.4% placebo-adjusted reduction in proteinuria compared to placebo at 34 weeks. 

• Rapid therapeutic onset, with significant proteinuria reduction observed as early as week 10 and sustained through 34 weeks. 

• A safety profile consistent with the established clinical history of ULTOMIRIS, with upper respiratory tract infections, nasopharyngitis, and infusion-related reactions reported as the most common adverse events. 

This approval further expands the rapidly growing arsenal of targeted therapies for rare, protein-spilling kidney diseases.  

With today’s approval, there are now nine FDA-approved treatment options for rare, protein-spilling kidney diseases, including therapies for IgAN, focal segmental glomerulosclerosis (FSGS), C3 glomerulopathy (C3G), primary IC-MPGN, and nephrotic syndrome. 

• EMPAVELI (pegcetacoplan) – Approved for C3G and primary IC-MPGN 
• FABHALTA (iptacopan) – Approved for C3G and IgAN 
• FILSPARI (esparsentán) - Approved for IgAN and FSGS 
• GAZYVA (obinutuzumab) – Approved for nephrotic syndrome 
• TARPEYO (budesonide) – Approved for IgAN 
• TRUTAKNA (atacicept) - Approved for IgAN 
• ULTOMIRIS (ravulizumab) – Approved for IgAN 
• VANRAFIA (atrasentan) – Approved for IgAN 
• VOYXACT (sibeprenlimab) - Approved for IgAN