Newly approved and emerging therapies are expanding treatment options and improving quality of life for people living with primary biliary cholangitis (PBC), according to a review published recently in Expert Opinion on Investigational Drugs. Patients who do not respond adequately to standard medication may soon have more effective, better-tolerated alternatives.
Ursodeoxycholic acid remains the first-line treatment for PBC, but as many as 40% of patients experience an incomplete response, leaving them at risk for ongoing liver injury and symptoms such as fatigue and severe itching. Until recently, the next option was obeticholic acid, but it was removed from U.S. and European markets due to safety concerns, underscoring the need for safer and more effective treatments.
Among the newest drugs are elafibranor and seladelpar, both belonging to a class known as PPAR agonists. In studies, elafibranor improved key liver markers that indicate reduced inflammation and bile buildup.
Seladelpar not only achieved similar liver protection but also significantly lessened pruritus (itching), one of the most distressing symptoms of PBC for many patients. These medications represent the first approved alternatives in years, offering hope for those who previously had limited options.
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A class of PPAR agonists called fibrates used to treat hyperlipidemia have shown promise in PBC. “Fibrates, particularly bezafibrate and fenofibrate, demonstrate biochemical and symptomatic benefits, but remain off-label treatments,” noted the review’s authors.
Other drugs under investigation may soon join the treatment landscape. Setanaxib, a NOX inhibitor, targets liver scarring by reducing oxidative stress. IBAT inhibitors such as linerixibat and odevixibat aim to ease itching by limiting bile acid reabsorption in the gut. Golexanolone, a compound that modulates brain neurotransmitters, is being studied for its potential to relieve fatigue, another common and debilitating symptom.
For patients, these developments could mean not only better lab results but also meaningful day-to-day improvements in comfort and function. The focus is shifting toward therapies that enhance overall well-being rather than simply normalize blood tests.
Experts emphasized the need for broader access to these medications and more research to identify which patients benefit most. As understanding of PBC evolves, treatment is moving toward a personalized approach that combines liver protection with quality-of-life gains, an encouraging shift for those living with this lifelong condition.
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