FDA Approves AstraZeneca’s Ultomiris for a Rare Kidney Disease

Patients with a rare disease that slowly destroys the kidneys have a new treatment option.

AstraZeneca said Wednesday that the U.S. Food and Drug Administration granted accelerated approval to its drug Ultomiris for adults with immunoglobulin A nephropathy, making it the first and only approved C5 inhibitor for the disease. A C5 inhibitor blocks one pathway of the immune system linked to kidney inflammation.

In the disease, an immune protein builds up in the kidney’s filtering tissue and causes inflammation. It progresses slowly and can end in kidney failure. More than 200,000 people in the United States have the condition, and about half of those with elevated urine protein levels or reduced kidney function are at high risk of kidney failure within 10 years, the company said.

The approval rests on an interim analysis of a late-stage trial. After 34 weeks, patients on Ultomiris had 43.4 percent less protein in their urine than patients on a placebo. Urine protein is a marker of how fast kidney damage is advancing.

Because the approval came through the accelerated pathway, it is not final. The trial continues through 106 weeks to test whether the drug slows the loss of kidney function itself, and that result will decide whether the approval becomes traditional. No new safety concerns were identified, the company said. Applications are also under review in Japan and other countries, according to Reuters.