US FDA grants accelerated approval to Ultomiris for IgA nephropathy
Regulators have cleared AstraZeneca’s Ultomiris for the reduction of proteinuria in adult patients with primary immunoglobulin A nephropathy.
The US Food and Drug Administration (FDA) has granted accelerated approval to AstraZeneca’s Alexion unit for the use of Ultomiris (ravulizumab) in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression. This marks the first time a C5 complement inhibitor has been approved for this specific renal condition.
Approval is based on a prespecified interim analysis from the phase III I CAN trial, which showed a 43.4% reduction in proteinuria for patients receiving the drug compared to placebo at week 34. While the data suggests a measurable physiological impact, regulators noted that it has not yet been established whether the drug definitively slows the decline of kidney function. Continued access is contingent upon further clinical trial results verifying the treatment's long-term benefit.
IgAN is a rare, progressive inflammatory disease affecting approximately 200,000 individuals in the US. The condition involves the abnormal deposition of IgA proteins in the kidneys, which triggers complement-driven inflammation and potential end-stage renal failure. Ultomiris, which is currently used for other rare diseases, is administered intravenously every eight weeks.