Novel preclinical developments of the primary sclerosing cholangitis treatment landscape

ABSTRACT

Introduction

Primary sclerosing cholangitis (PSC) is a chronic cholestatic liver disease associated with inflammation, fibrosis, and destruction of intra- and extrahepatic bile ducts. Despite substantial recent advances in our understanding of PSC, the only proven treatment of PSC is liver transplantation. There is an urgent unmet need to find medical therapies for this disorder.

Areas covered

Multiple drugs are currently under evaluation as therapeutic options for this disease. This article summarizes the literature on the various novel therapeutic options that have been investigated and are currently under development for the treatment of PSC.

Expert opinion

In the next decade, more than one drug will likely be approved for the treatment of the disease, and we will be looking at combination therapies for the optimal management of the disease.

KEYWORDS:

• Primary sclerosing cholangitis
• treatment
• investigational drugs
• clinical trials
• landscape

Article highlights

• Primary sclerosing cholangitis (PSC) is a rare cholestatic liver disorder which can progress to advanced liver disease.

• Liver transplantation is curative; however, its use is reserved for patients with advanced liver disease.

• Currently, there is no FDA approved treatment for the management of PSC, however multiple drugs are currently under investigation as potential treatment.

• These drugs are in different phases of investigation and is likely that soon there will be a treatment option for this rare disorder.

• In the next decade, it is likely that more than one drug will be approved for this condition, and we will be evaluating combination therapies for the management of the disease.

Declaration of interests

KV Kowdley declares research support from CymaBay Therapeutics; grants and/or contracts from 89Bio, Genfit, Gilead, GlaxoSmithKline, Hanmi, HighTide, Intercept, Madrigal, Mirum, NGM, Pfizer, Pliant, and Viking; royalties/licenses from UpToDate; consulting fees from 89Bio, Calliditas Therapeutics, CymaBay Therapeutics, Genfit, Gilead, Inipharm, Intercept, Madrigal, Mirum, NGM, and Pliant; payment/honoraria for lectures, presentations, speakers bureaus, manuscript writing, or educational events from AbbVie, Gilead, and Intercept; participation in a data safety monitoring board or advisory board for CTI, Medpace and Labcorp; stock or stock options for Inipharm; and receipt of equipment, materials, drugs, medical writing, gifts, or other services from Sonic Insight. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.