AstraZeneca's rare disease unit Alexion has secured accelerated US approval for Ultomiris in immunoglobulin A nephropathy (IgAN), a rare inflammatory kidney condition that can lead to organ failure.
The drug, known generically as ravulizumab, becomes the first and only C5 complement inhibitor approved to reduce proteinuria, the presence of excess protein in urine, in adults with the disease.
IgAN affects more than 200,000 people in the US, with around half of those with elevated protein levels or reduced kidney function at high risk of progressing to kidney failure within 10 years of diagnosis.
The approval is based on a prespecified interim analysis of the Phase III I CAN trial, in which Ultomiris delivered a 43.4% reduction in proteinuria versus placebo at 34 weeks.
The reduction was observed as early as week 10 and was sustained through the 34-week analysis, with consistent results across patient subgroups.
The approval is conditional, and continued authorisation will depend on verification of clinical benefit through the ongoing I CAN trial, which will assess changes in estimated glomerular filtration rate (eGFR), a measure of kidney function, at week 106.
Regulatory submissions based on the interim results are also under review in Japan and additional countries.
Ultomiris is already approved across four other indications, including paroxysmal nocturnal haemoglobinuria and generalised myasthenia gravis, making IgAN its fifth approved use.
The drug is administered intravenously every eight weeks in adults following a loading dose, offering a longer dosing interval than some existing treatments.
Marc Dunoyer, chief executive of Alexion, said the approval underscored the need for more targeted therapies for patients who continued to face the risk of progression to kidney failure despite available treatments.
