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New Therapies on the Horizon for Primary Biliary Cholangitis

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Abstract

Primary biliary cholangitis (PBC) is a chronic cholestatic liver disease that can progress to cirrhosis and hepatic failure if left untreated. Ursodeoxycholic acid (UDCA) was introduced as a first-line drug for PBC around 1990; it remarkably improved patient outcomes, leading to the nomenclature change of PBC in 2015, from primary biliary “cirrhosis” to primary biliary “cholangitis.” Nevertheless, 20–30% of patients exhibit an incomplete response to UDCA, resulting in significantly worse outcomes compared to those with a complete response. Therefore, improving the long-term outcomes of patients with an incomplete response to UDCA has been recognized as an unmet need. In addition, patients with PBC often suffer from a variety of debilitating symptoms, such as pruritus, fatigue and sicca syndrome, which significantly impair their health-related quality of life. Thus, appropriate management of these symptoms is currently regarded as another unmet need for PBC treatment. In this review, several compounds and drugs under clinical trials that can potentially solve these unmet needs are comprehensively discussed, and future directions of treatment policy of PBC are proposed for significantly improving long-term outcome as well as health-related quality of life of patients.

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  1. Department of Medicine, Teikyo University School of Medicine, 2-11-1, Kaga, Itabashi-ku, Tokyo, 173-8605, Japan

    Atsushi Tanaka

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This work was supported by a Health and Labor Sciences Research Grant (research on intractable hepatobiliary disease) issued by the Ministry of Health, Labor, and Welfare of Japan (Grant Number JPMH20FC1023). The sponsor had no role in the study.

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Dr. A. Tanaka reports receiving consultant fees from EA Pharma, GlaxoSmithKline, Kowa Company Ltd., and Gilead Sciences.

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Tanaka, A. New Therapies on the Horizon for Primary Biliary Cholangitis. Drugs 84, 1–15 (2024). https://doi.org/10.1007/s40265-023-01979-1

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