What are the new therapeutic treatments for Primary Biliary Cholangitis (PBC)?

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Last updated: April 10, 2025 • View editorial policy

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From the Guidelines

The most effective new therapeutic treatment for primary biliary cholangitis (PBC) is obeticholic acid (OCA) at a dose of 5-10 mg daily, which has been shown to improve liver biochemistry and reduce bile acid production and inflammation 1.

Treatment Options

  • Ursodeoxycholic acid (UDCA) remains the first-line therapy at 13-15 mg/kg/day, which improves liver biochemistry and delays disease progression.
  • For patients with inadequate response to UDCA, OCA is now approved as a second-line treatment.
  • Bezafibrate, a PPAR agonist, can be added at 400 mg daily as combination therapy with UDCA for those with incomplete response, though it's not FDA-approved for PBC in the US.

Emerging Therapies

  • Seladelpar, a PPAR-delta agonist, shows promise in clinical trials.
  • Elafibranor, another PPAR agonist, is also being investigated as a potential treatment for PBC.

Symptom Management

  • Cholestyramine can be used to manage pruritus.
  • Structured exercise programs can help address fatigue.

Monitoring

  • Regular monitoring of liver function tests every 3-6 months is essential to assess treatment response and disease progression. It's worth noting that the use of OCA in pregnancy or during lactation is not recommended due to a lack of safety data 1. Additionally, bezafibrate has been shown to be effective in alleviating moderate to severe itch in people with PSC and PBC treated with UDCA 1.

From the FDA Drug Label

OCALIVA, a farnesoid X receptor (FXR) agonist, is indicated for the treatment of adult patients with primary biliary cholangitis (PBC) without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension, either in combination with ursodeoxycholic acid (UDCA) with an inadequate response to UDCA or as monotherapy in patients unable to tolerate UDCA. This indication is approved under accelerated approval based on a reduction in alkaline phosphatase (ALP). An improvement in survival or disease-related symptoms has not been established. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

New Therapeutic Treatment for PBC:

  • Obeticholic acid (PO) is a new therapeutic treatment for primary biliary cholangitis (PBC)
  • It is indicated for adult patients with PBC without cirrhosis or with compensated cirrhosis who do not have evidence of portal hypertension
  • The treatment can be used in combination with ursodeoxycholic acid (UDCA) or as monotherapy in patients unable to tolerate UDCA 2, 2, 2.
  • The approval is based on a reduction in alkaline phosphatase (ALP), but an improvement in survival or disease-related symptoms has not been established.

From the Research

New Therapeutic Treatments for Primary Biliary Cholangitis (PBC)

  • Primary biliary cholangitis (PBC) is a chronic, progressive autoimmune liver disease that can lead to the destruction of the small bile ducts, resulting in cholestasis and intrahepatic cholangitis 3, 4, 5, 6, 7.
  • Ursodeoxycholic acid (UDCA) is currently the primary medication for the treatment of PBC, but some patients do not respond adequately to UDCA, putting them at an increased risk of disease progression 3, 4, 5, 6, 7.

Alternative Therapies for UDCA Non-Responders

  • Obeticholic acid (OCA) has been shown to be effective in improving surrogate markers of prognosis in PBC, including a significant decrease in aspartate aminotransferase (AST), alanine aminotransferase (ALT), and gamma-glutamyl transferase (GGT) 4, 5.
  • Bezafibrate, a PPAR agonist, has also been shown to improve liver biochemistries and reduce the risk of disease progression in patients with PBC who are non-responders to UDCA 3, 4, 6.
  • Combination therapy with UDCA and OCA or bezafibrate may be considered in patients showing inadequate response to monotherapy 4, 6.

Efficacy and Safety of PPAR Agonists

  • A systematic review and meta-analysis of randomized controlled trials found that PPAR agonists, including bezafibrate, effectively reduce alkaline phosphatase (ALP), GGT, and total bilirubin levels in patients with PBC 6.
  • The use of PPAR agonists in combination with UDCA has been shown to be safe and well-tolerated, with no significant increase in adverse events compared to UDCA alone 3, 6.

Future Directions

  • Further investigation is needed to provide high-quality evidence to formulate standardized therapies for patients with PBC who are non-responders to UDCA 4, 7.
  • Randomized controlled trials are necessary to confirm the efficacy and safety of alternative therapies, including OCA and PPAR agonists, in patients with PBC 4, 7.

References

1
Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

4
Research

Primary biliary cholangitis: treatment.

Current opinion in gastroenterology, 2021

7
Research

Treatment of primary biliary cholangitis ursodeoxycholic acid non-responders: A systematic review.

Liver international : official journal of the International Association for the Study of the Liver, 2017

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Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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