GSK plc (LSE/NYSE: GSK) today announced the US Food and Drug Administration (FDA) has accepted for review the NDA for linerixibat, an investigational targeted inhibitor of the ileal bile acid transporter (IBAT), for the treatment of cholestatic pruritus in patients with PBC, a rare autoimmune liver disease.

GSK plc（LSE/NYSE：GSK）今天宣布，美国食品药品监督管理局（FDA）已接受审查 linerixibat 的新药申请（NDA）。Linerixibat 是一种研究性回肠胆汁酸转运蛋白（IBAT）靶向抑制剂，用于治疗原发性胆汁性胆管炎（PBC）患者的相关瘙痒症，这是一种罕见的自身免疫性肝病。

The Prescription Drug User Fee Act (PDUFA) goal date is 24 March 2026..

《处方药使用者费用法案》（PDUFA）的目标日期是2026年3月24日。

Kaivan Khavandi, SVP, Global Head, Respiratory, Immunology & Inflammation R&D, GSK, said:

凯文·哈万迪，GSK全球呼吸、免疫与炎症研发高级副总裁兼全球负责人表示：

“The FDA’s acceptance of this file is an important milestone in the development of linerixibat. We believe that linerixibat has the potential to make a difference in the lives of patients living with relentless itch associated with PBC and its related sleep interference. These are debilitating symptoms which currently have very limited treatment options.”.

“FDA对该文件的接受是linerixibat开发过程中的一个重要里程碑。我们相信linerixibat有潜力改变与PBC相关的持续瘙痒及其相关睡眠干扰患者的生活。这些症状令人虚弱，目前的治疗选择非常有限。”

The application is based on positive data from the GLISTEN phase III trial, presented in May at the European Association for the Study of the Liver (EASL) Congress. GLISTEN met both primary and key secondary endpoints demonstrating a rapid, significant and sustained improvement in cholestatic pruritus and itch-related sleep interference versus placebo.

该申请基于 GLISTEN 三期试验的积极数据，该数据于 5 月在欧洲肝病研究协会 (EASL) 大会上公布。GLISTEN 达到了主要和关键次要终点，证明与安慰剂相比，在胆汁淤积性瘙痒和与瘙痒相关的睡眠干扰方面有快速、显著且持续的改善。

The safety profile of linerixibat was consistent with previous studies and the mechanism of IBAT inhibition..

利那西巴特的安全性特征与以往的研究和IBAT抑制机制一致。

Linerixibat is currently not approved anywhere in the world.

Linerixibat 目前在全球任何地方都未获批准。

About cholestatic pruritus in PBC

关于PBC中的胆汁淤积性瘙痒

In PBC, a cholestatic liver disease, bile flow from the liver is disrupted. The resulting excess bile acids in circulation are thought to play a causal role in cholestatic pruritus, an internal itch that cannot be relieved by scratching. Pruritus can occur at any stage of PBC disease or biochemical control, and is experienced in varying degrees of severity by up to 90% of people living with PBC..

在原发性胆汁性胆管炎（PBC）中，这是一种胆汁淤积性肝病，肝脏的胆汁流动受到阻碍。由此导致的循环中过量胆汁酸被认为在胆汁淤积性瘙痒中起因果作用，这种内部瘙痒无法通过抓挠缓解。瘙痒可以在PBC疾病或生化控制的任何阶段发生，并且多达90%的PBC患者会经历不同程度的严重瘙痒。

The first line treatment for PBC controls disease in approximately 70% of patients, but does not reduce the severity or impact of the pruritus.

原发性胆汁性胆管炎的一线治疗可控制大约70%患者的病情，但并不能减轻瘙痒的严重程度或影响。

Cholestatic pruritus is a serious condition that can be debilitating, with patients experiencing sleep disturbance, fatigue, impaired quality of life and even sometimes requiring liver transplantation in the absence of liver failure.

胆汁淤积性瘙痒是一种严重的疾病，可能会导致患者虚弱，出现睡眠障碍、疲劳、生活质量下降，甚至有时在没有肝衰竭的情况下需要进行肝移植。

About linerixibat (GSK2330672)

关于linerixibat（GSK2330672）

Linerixibat is an IBAT inhibitor, a targeted oral agent with potential to treat cholestatic pruritus (itch) associated with the rare autoimmune liver disease known as PBC. By inhibiting bile acid re-uptake, linerixibat reduces multiple mediators of pruritus in circulation. The US Food and Drug Administration and the European Medicines Agency have granted orphan drug designation for linerixibat in the treatment of cholestatic pruritus in patients with PBC..

Linerixibat是一种IBAT抑制剂，是一种有针对性的口服药物，有潜力治疗与罕见的自身免疫性肝病（称为原发性胆汁性胆管炎，PBC）相关的胆汁淤积性瘙痒（痒）。通过抑制胆汁酸再摄取，linerixibat减少循环中多种瘙痒介质。美国食品药品监督管理局和欧洲药品管理局已授予linerixibat在治疗PBC患者胆汁淤积性瘙痒方面的孤儿药资格。

About the GLISTEN trial

关于GLISTEN试验

GLISTEN is a double-blind, randomised, placebo-controlled, phase III trial (NCT04950127; GSK study 212620) conducted in 238 PBC patients with cholestatic pruritus initially enrolled equally into active and placebo arms (n=119 each). The primary analysis evaluated the efficacy and safety of linerixibat compared with placebo.

GLISTEN 是一项双盲、随机、安慰剂对照的 III 期临床试验（NCT04950127；GSK 研究 212620），最初将 238 名胆汁淤积性瘙痒的原发性胆管炎（PBC）患者平均分配至活性药物组和安慰剂组（各 119 名）。主要分析评估了 linerixibat 与安慰剂的疗效和安全性。

Participants with moderate to severe itch were enrolled. Participants initially received either linerixibat or placebo and had the potential to cross over in a part B of the trial. Primary and secondary outcome measures were assessed using a 0-10 numerical rating scale for worst itch and itch-related sleep interference.

中度至重度瘙痒的参与者被纳入研究。参与者最初接受 linerixibat 或安慰剂治疗，并在试验的 B 阶段有可能交叉。主要和次要结局指标通过 0-10 数字评分量表评估最严重瘙痒和瘙痒相关的睡眠干扰。

Stable use of guideline suggested anti-itch therapy was permitted. The trial was the first truly global PBC study completed in 19 countries including the Americas, Europe, China and Japan..

允许稳定使用指南建议的止痒疗法。该试验是第一个真正全球性的PBC研究，在包括美洲、欧洲、中国和日本等19个国家完成。

About GSK research in hepatology

关于GSK在肝病学领域的研究

GSK is currently investigating multiple potential treatments for patients with liver disease. In addition to PBC, we are also investigating potential treatments for chronic hepatitis B, alcohol-related liver disease (ALD), and metabolic dysfunction-associated steatohepatitis (MASH).

GSK目前正在研究多种针对肝病患者的潜在治疗方法。除了PBC，我们还在研究针对慢性乙型肝炎、酒精相关性肝病（ALD）以及代谢功能障碍相关脂肪性肝炎（MASH）的潜在治疗方法。

About GSK

关于GSK

GSK is a global biopharma company with a purpose to unite science, technology, and talent to get ahead of disease together. Find out more at gsk.com.

GSK是一家全球生物制药公司，致力于将科学、技术和人才结合在一起，共同战胜疾病。欲了解更多信息，请访问gsk.com。