The impact of deep response to ursodeoxycholic acid in primary biliary cholangitis – should it be the new clinical standard?

Purpose of review

This review explores the emerging concept of “deep response” in primary biliary cholangitis (PBC), defined by the normalization of biochemical markers, particularly alkaline phosphatase and bilirubin. It examines its potential as a new standard for disease management and its implications for long-term patient outcomes, health policies, and clinical decision-making.

Recent Findings

Recent studies suggest that achieving a deep response significantly improves long-term outcomes in some patients with PBC. In particular, a significant complication-free survival gain was observed among patients who at baseline were at high risk for disease progression. However, limitations in data and the variability in patient populations pose challenges for universal adoption of this standard.

Summary

Deep biochemical response represents a promising new standard for optimizing PBC management, offering measurable goals for clinicians and potentially improved long-term outcomes for patients. However, further research is necessary to better define the appropriate biochemical thresholds, understand the risks of overprescribing, and identify patient subgroups that are most likely to benefit from this strategy. A balanced, patient-centered approach incorporating deep response into comprehensive management could improve care for high-risk PBC patients.

Current Opinion in Gastroenterology - Copyright © 2025 Wolters Kluwer Health, Inc. All rights reserved.

Note: Obeticholic acid, marketed under the brand name Ocaliva^® for the treatment of primary biliary cholangitis (PBC), was voluntarily withdrawn from the US market by Intercept Pharmaceuticals following a request from the US Food and Drug Administration (FDA) on 11/14/2025