FDA Grants Accelerated Approval to Ultomiris for Rare Kidney Disease; Proteinuria Cut by 43.4% in Trial
AstraZeneca said on Wednesday, October 21, that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to its treatment Ultomiris for adults with immunoglobulin A nephropathy, a rare kidney disease that can lead to renal failure, Reuters reported.
The company said Ultomiris reduced proteinuria — excess protein in the urine — by 43.4 percent compared with placebo after 34 weeks in an interim analysis of a late-stage trial. The drug is the first and only approved C5 inhibitor for the disease, targeting an immune-system pathway linked to kidney inflammation. More than 200,000 people in the United States have the condition, according to the company, 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 approval was granted under the FDA's accelerated pathway. The ongoing trial will continue through 106 weeks to determine whether Ultomiris slows the loss of kidney function to support traditional approval. The company said no new safety concerns were identified in the study, and applications are also under review in Japan and other countries.
Published by GSM · USA (original Chinese edition)
Photo: 图：肾脏 CT 影像（资料图）。维基共享资源，CC BY 4.0。
