New therapies for primary biliary cholangitis.

BACKGROUND

Primary Biliary Cholangitis (PBC) is a cholestatic autoimmune liver disease of small bile ducts. Ursodeoxycholic acid (UDCA) was approved as a first line therapeutic option for PBC in 1997. It was later noted that 40% of patients with PBC are either non-responders or intolerant to UDCA. In 2016, obeticholic acid (OCA) was approved as a second-line therapy for PBC. However, due to the side effects associated with OCA and the FDA restricting its use in patients with cirrhosis, there was a need for additional therapies.

AIMS: This review summarizes the current literature regarding the new and emerging therapies for patients with PBC.

KEY FINDINGS: In 2024, two new therapies, elafibranor and seladelpar were approved as a second-line treatment for PBC. In 2025 OCA was withdrawn from the market. Multiple additional therapies targeting biochemical remission are under investigation. Furthermore, a new class of medication, ileal bile acid transporter inhibitors (IBAT inhibitors), is being studied for pruritus among these patients.

CONCLUSION

The therapeutic landscape for PBC has been rapidly evolving with the discovery of second-line agents. Ongoing trials studying biochemical response, symptom control and long-term clinical outcomes among patients with PBC will be beneficial.