15 hours ago
Novel Therapies for Primary Biliary Cholangitis
Primary biliary cholangitis (PBC) is a chronic, progressive autoimmune disease characterized by ongoing bile duct inflammation and elevated alkaline phosphatase (ALP). Ursodeoxycholic acid (UDCA) is well established as the initial treatment for PBC. This medication, which helps move bile through the liver, can improve liver function and reduce scarring of the liver. However, UDCA does not appear to provide significant improvement in itching or fatigue.
In this ReCAP, Dr Marlyn Mayo of the University of Texas Southwestern Medical Center, Dallas, Texas, discusses novel therapies for PBC in patients not responding to or tolerating UDCA. She notes that obeticholic acid (OCA), the initial second-line therapy approved in 2016, is associated with increased itching, no fatigue improvement, and rare instances of liver worsening — emphasizing the need for safer, more effective second-line options.
The approval of PPAR agonists seladelpar and elafibranor in 2024 marked a significant development, according to Dr Mayo. Both compounds demonstrated strong biochemical improvements in their phase 3 trials, effectively lowering ALP levels. A key advantage over OCA is that these PPAR agonists enhance tolerability by alleviating itching and appear to reduce fatigue, a previously untreatable symptom in PBC.
