美国食品药品监管局批准首款原发性膜性肾病药物 Gazyva，三期研究完全缓解率 36% 对 6%
美国食品药品监管局（FDA）2026年11月13日批准 Gazyva（obinutuzumab）注射液，用于治疗有疾病进展风险的成人原发性膜性肾病患者、诱导蛋白尿缓解。这是首款专门获批用于成人原发性膜性肾病的药物。Gazyva 此前已获批用于治疗某些癌症、活动性狼疮性肾炎与儿童期起病的特发性肾病综合征。
原发性膜性肾病是一种罕见肾病，由免疫系统攻击肾小球引起。肾小球是肾脏内负责过滤血液废物的微小滤器，受损发炎后会把大量蛋白漏入尿液，患者可出现高胆固醇、高血压、严重水肿、乏力、肾衰竭等并发症。
疗效与安全性来自 MAJESTY 三期研究。这是一项随机、对照、开放标签、多中心研究，共纳入 142 名成人原发性膜性肾病患者，在 104 周主研究期内分别接受 Gazyva 或他克莫司治疗，他克莫司是临床上有时超说明书用于该病的另一种治疗。主要终点为第 104 周达到蛋白尿完全缓解的患者比例，完全缓解定义为尿蛋白肌酐比不高于 0.3 g/g、估算肾小球滤过率稳定，且未接受挽救治疗、未出现治疗失败或提前退出研究。结果显示，Gazyva 组完全缓解率为 36%，他克莫司组为 6%，差异显著。
安全性方面，Gazyva 标签带有乙型肝炎病毒再激活与进行性多灶性白质脑病（PML）的黑框警告，PML 是免疫功能低下者可能发生的罕见严重脑部病毒感染。该研究中最常见的不良反应为输液相关反应与上呼吸道感染，完整安全信息见药品说明书。
此次批准同时获得突破性疗法、孤儿药与优先审评三项资格认定。（来源：美国食品药品监管局）
FDA Approves Gazyva, First Drug for Primary Membranous Nephropathy, With 36% vs 6% Complete Remission in Phase 3 Trial
The U.S. Food and Drug Administration (FDA) on November 13, 2026 approved Gazyva (obinutuzumab) injection to treat adults with primary membranous nephropathy at risk of disease progression, to induce remission of proteinuria. It is the first drug approved specifically for the treatment of adults with primary membranous nephropathy. Gazyva was previously approved for treating certain cancers, active lupus nephritis, and childhood-onset idiopathic nephrotic syndrome.
Primary membranous nephropathy is a rare kidney disease caused by immune system attack on the glomeruli, the tiny filters in the kidneys that clean waste products from the blood. The glomeruli become inflamed and damaged, leaking large amounts of protein into the urine. Patients can develop high cholesterol, high blood pressure, severe swelling, fatigue, kidney failure, and other complications.
Efficacy and safety were evaluated in MAJESTY, a Phase 3, randomized, controlled, open-label, multicenter study in 142 adults with primary membranous nephropathy. During the main study period of 104 weeks, patients were randomly assigned to receive either Gazyva or tacrolimus, another treatment sometimes used off-label for the disease. The primary endpoint was the proportion of patients who achieved complete remission of proteinuria at week 104, defined as a urine protein-creatinine ratio of no more than 0.3 g/g with a stable estimated glomerular filtration rate, without receipt of rescue therapy, treatment failure, or early study discontinuation. The proportion achieving complete remission was 36% in the Gazyva group compared with 6% in the tacrolimus group, a significant difference.
Gazyva's label carries a boxed warning for hepatitis B virus reactivation and progressive multifocal leukoencephalopathy (PML), a rare and serious brain infection caused by a virus in people with a weakened immune system. The most common side effects in the study were infusion-related reactions and upper respiratory tract infection. See the full prescribing information for complete safety details.
The approval received Breakthrough Therapy, Orphan Drug, and Priority Review designations. (Source: U.S. Food and Drug Administration)
来源：美国食品药品监管局
发布：GSM·美国（发布链接）
图片来源：图：美国食品药品监管局（FDA）总部大楼（固定图库联邦核心包）。 / Photo: Headquarters of the U.S. Food and Drug Administration (fixed federal-core asset-pack image).
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