New treatment for primary biliary cholangitis

20 January, 2025

Primary biliary cholangitis is a rare liver disease causing bile duct damage. New therapies like seladelpar improve outcomes for treatment-resistant patients, offering better symptom management.

Primary biliary cholangitis is a liver disease in which the bile ducts (tubes inside the liver) become inflamed and eventually destroyed. The natural evolution of this disease is the development of liver cirrhosis. Primary biliary cholangitis is considered a rare disease since it affects <50 people per 100,000.

Primary biliary cholangitis causes fatigue and often intense pruritus (generalized itching throughout the body). In the final stages, if untreated, jaundice (yellowing of the skin and the whites of the eyes) and liver failure may occur.

Currently, the disease has no cure, but it can be inactivated with very effective drugs that control its natural evolution and prognosis. Ursodeoxycholic acid is the drug of choice for treatment. If this medication does not achieve normalization of the analysis, another one (bezafibrate) is added. If remission is not achieved despite this (30-40% of non-responders), another drug, obeticholic acid, can be added. Despite this triple therapy, there are patients with primary biliary cholangitis who do not respond, which is why new medications are being developed.

Use of Seladelpar like treatment

Seladelpar is a new drug for the treatment of refractory primary biliary cholangitis that has already been approved for use in the United States. Recently, the results of a study were reported in 337 patients with primary biliary cholangitis who had not responded to ursodeoxycholic acid and who received 10 mg of seladelpar daily orally. At 12, 24 and 30 months of treatment, 73%, 73% and 81% of the patients, respectively, presented a biochemical response and the patients’ pruritus also improved.

In conclusion, this drug can be very useful to treat those patients who have not responded or cannot tolerate ursodeoxycholic acid. In the opinion of Dr. Carreño and the Foundation’s doctors, it would also be interesting to study the effectiveness of seladelpar in patients resistant to bezafibrate and/or obeticholic acid.

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