FDA批准扩大诺华罕见病药物的使用范围，作为唯一治疗罕见肾病的方法-动脉网

FDA Approves Expanded Use Of Novartis Rare Disease Drug As Only Treatment For Rare Type Of Kidney Disease

benzinga 等信源发布 2025-03-21 11:36



可切换为仅中文







On Thursday, the U.S. Food and Drug Administration (FDA) approved

周四，美国食品和药物管理局 (FDA) 批准了

Novartis AG’s

诺华公司

NVS

oral Fabhalta (iptacopan) for adults with C3 glomerulopathy (C3G) to reduce proteinuria (protein in the urine), making it the first and only treatment

口服Fabhalta（iptacopan）用于治疗患有C3肾小球病（C3G）的成人，以减少蛋白尿（尿液中的蛋白质），成为首个也是唯一一个治疗方法。

approved for this condition

批准用于此病症

。

C3G is a rare kidney disease characterized by damage to the glomeruli (kidney filters) due to dysregulation of the complement system, specifically the alternative pathway, leading to C3 protein accumulation.

C3G是一种罕见的肾脏疾病，其特征是由于补体系统（特别是替代途径）失调导致肾小球（肾脏过滤器）受损，进而引起C3蛋白积累。

Also Read:

另请阅读：

Novartis New Data From Single-Dose Gene Therapy Shows Meaningful Efficacy, Safety In Patients With Spinal Muscular Atrophy

诺华单剂量基因疗法新数据显示对脊髓性肌萎缩症患者有显著疗效和安全性

The pivotal Phase 3 APPEAR-C3G study

关键的第3阶段APPEAR-C3G研究

evaluated

评估过的

the efficacy and safety of twice-daily oral Fabhalta in adult patients with C3G.

每日两次口服Fabhalta对C3G成年患者的疗效和安全性。

Treatment with Fabhalta resulted in a clinically meaningful reduction in proteinuria, which was seen as early as 14 days and sustained at 12 months.

Fabhalta治疗导致蛋白尿显著减少，这种效果最早在14天时出现，并在12个月时仍然维持。

Similarly, in the open-label period, proteinuria reduction was seen in participants who switched to Fabhalta.

同样，在开放标签期内，换用Fabhalta的参与者出现了蛋白尿减少的情况。

Last month, Fabhalta received a positive CHMP Opinion in C3G by the European Medicines Agency (EMA). Regulatory reviews for this indication are ongoing in China and Japan.

上个月，Fabhalta 在欧洲药品管理局 (EMA) 的 C3G 适应症中获得了积极的 CHMP 意见。该适应症在中国和日本的监管审查仍在进行中。

This is the third US approval for Fabhalta and its second within the Novartis kidney disease portfolio since August 2024, when Fabhalta was granted accelerated approval by the FDA for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression..

这是Fabhalta获得的第三次美国批准，也是自2024年8月以来诺华肾病产品组合中的第二次批准。当时，Fabhalta被FDA加速批准用于减少原发性免疫球蛋白A肾病（IgAN）且有快速疾病进展风险的成年患者的蛋白尿。

Continued approval for this indication is contingent upon confirmatory evidence.

该适应症的持续批准取决于确证性证据。

Fabhalta received its first FDA approval in December 2023 for adults with paroxysmal nocturnal hemoglobinuria (PNH).

Fabhalta 于 2023 年 12 月首次获得 FDA 批准，用于治疗患有阵发性夜间血红蛋白尿 (PNH) 的成人。

In the fourth quarter of 2024, Fabhalta generated

2024年第四季度，Fabhalta公司生成了

sales of $57 million

5700万美元的销售额

。

In addition to Fabhalta, Novartis is advancing the late-stage development of two additional IgAN therapies with highly differentiated mechanisms of action: atrasentan, an investigational oral endothelin A receptor antagonist that received FDA filing acceptance in Q2 2024 with a decision anticipated in H1 2025, and zigakibart, an investigational subcutaneously administered anti-APRIL monoclonal antibody currently in Phase 3 development. .

除Fabhalta外，诺华还在推进另外两种IgAN疗法的晚期开发，这些疗法具有高度差异化的机制：atrasentan，一种研究中的口服内皮素A受体拮抗剂，于2024年第二季度获得FDA受理，预计2025年上半年做出决定；以及zigakibart，一种研究中的皮下注射抗APRIL单克隆抗体，目前处于三期临床开发阶段。

Price Action:

价格行为:

NVS stock is down 0.45% at $112.70 at the last check Friday.

NVS股票在上周五最后一次检查时下跌了0.45%，至112.70美元。

Read Next:

接下来阅读：

Johnson & Johnson To Invest $55 Billion In US To Boost Manufacturing, Drug Discovery

强生公司将投资550亿美元用于提升美国的制造业和药物研发。

Photo by Taljat David via Shutterstock

照片由塔尔亚特·大卫通过 Shutterstock 提供

NVS

非易失性存储器 (Non-Volatile Storage)