食品药品监管局 FDA 加速批准 Ultomiris 用于罕见肾病：IgA 肾病患者蛋白尿降幅达43.4%
据路透社10月21日报道，AstraZeneca 当日宣布，美国食品药品监管局 FDA 已加速批准其药物 Ultomiris 用于治疗成人免疫球蛋白A肾病（IgA 肾病）。这是一种可导致肾衰竭的罕见肾病。
公司称，在后期试验的中期分析中，治疗34周后，Ultomiris 使患者蛋白尿（尿液中蛋白质过多）较安慰剂下降43.4%。该药是首个、也是目前唯一获批用于这一疾病的 C5 抑制剂，作用于一条与肾脏炎症相关的免疫通路。公司称，美国有超过20万人患有此病，其中约半数尿蛋白升高或肾功能下降的患者在10年内面临肾衰竭高风险。
此次批准走的是加速批准通道。相关试验将继续进行至106周，以确认 Ultomiris 能否延缓肾功能丧失、支持转为传统批准。公司称研究中未发现新的安全问题；该药的上市申请也正在日本等其他国家接受审评。
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.
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图片来源：图：肾脏 CT 影像（资料图）。维基共享资源，CC BY 4.0。
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