Primary Biliary Cholangitis and Primary Sclerosing Cholangitis Therapy Landscape

Area of Science:

• Hepatology
• Gastroenterology
• Internal Medicine

Background:

• Primary biliary cholangitis (PBC) and primary sclerosing cholangitis (PSC) are rare chronic cholestatic liver diseases.
• These conditions can lead to cirrhosis and liver failure if untreated.
• Current treatment goals include halting disease progression and alleviating associated symptoms.

Purpose of the Study:

• To review the current and emerging treatment landscape for PBC and PSC.
• To highlight recent advancements and ongoing investigations in therapy development.

Main Methods:

• Literature review of current and upcoming treatments for PBC and PSC.
• Analysis of approved therapies and investigational agents.

Main Results:

• Ursodeoxycholic acid is the historical first-line PBC treatment; obeticholic acid and fibrates are second-line/adjunctive.
• Two new second-line agents for PBC recently gained FDA approval.
• Several promising therapies for PBC are under investigation.
• Liver transplantation is the only approved treatment for PSC, but emerging therapies show promise.

Conclusions:

• The treatment landscape for PBC is rapidly expanding with new approved and investigational agents.
• While PSC treatment options are limited, emerging therapies offer encouraging results.
• Further research is crucial for developing effective treatments for both rare cholestatic diseases.