What Did the Research Discover?
Primary biliary cholangitis (PBC) is a rare liver disease that gradually damages the bile ducts, leading to liver scarring. If left untreated, it can progress to cirrhosis and severe liver disease. The current first-line treatment, ursodeoxycholic acid (UDCA), does not work for up to 40% of patients, and the second-line option, obeticholic acid, is not always effective and can cause severe itching. Researchers conducted a study to determine whether elafibranor, a new oral medication, could help patients with PBC who did not respond well to UDCA.
This phase 3 clinical trial included 161 patients from 14 countries. The patients were randomly assigned to receive either elafibranor (80 mg daily) or a placebo for 52 weeks. The researchers measured success based on whether patients had a significant reduction in alkaline phosphatase (ALP) levels, a key indicator of liver health. They also looked at changes in itching severity, which is a common symptom of PBC.
At the end of the study, 51% of patients taking elafibranor met the criteria for biochemical improvement, compared to only 4% of those on placebo. Additionally, 15% of patients in the elafibranor group had completely normal ALP levels, while no patients in the placebo group achieved this. However, elafibranor did not significantly reduce itching in patients with moderate-to-severe symptoms. Some patients experienced side effects such as abdominal pain, diarrhea, nausea, and vomiting, though no severe safety concerns were reported.
How Can I Apply This Information?
This study suggests that elafibranor may be an effective second-line treatment for patients with primary biliary cholangitis who do not respond well to UDCA. It significantly improved key markers of liver function and had a manageable safety profile. However, it did not show a major impact on itching, a symptom that can greatly affect quality of life for PBC patients.
If you have PBC and are not responding well to current treatments, you may want to discuss elafibranor with your doctor. As of June 2024, elafibranor was approved by the FDA under accelerated approval for PBC. It is indicated for use in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
Source:
Kowdley KV, Bowlus CL, Levy C, Akarca US, Alvares-da-Silva MR, Andreone P, Arrese M, Corpechot C, Francque SM, Heneghan MA, Invernizzi P. Efficacy and safety of elafibranor in primary biliary cholangitis. New England Journal of Medicine. 2024 Feb 29;390(9):795-805. https://www.nejm.org/doi/full/10.1056/NEJMoa2306185
