The drug elafibranor appears to be effective and safe for the treatment of primary biliary cholangitis (PBC), significantly improving bile flow throughout the biliary system, according to a study in the New England Journal of Medicine.
PBC is characterized by the destruction of a network of tubes that transports bile outside the liver (intrahepatic bile ducts), by the immune system. Intrahepatic bile duct destruction leads to bile accumulation within the liver (cholestasis), which in turn leads to liver damage that can produce end stage liver disease.
The most common laboratory tests used to measure cholestasis are alkaline phosphatase (AP) and bilirubin. High levels of any of these markers in a blood test are indicative of cholestasis.
Currently, the available medications for PBC have limited efficacy in a significant percentage of patients. They are associated with severe adverse effects that can lead to treatment discontinuation in up to 5% of cases.
Elafibranor decreases inflammation caused by bile accumulation.The authors aimed to assess its efficacy in reducing bilirubin, and AP levels in patients with PCB compared to those receiving a placebo and determine its safety profile.
Results from the study including over 160 patients showed that after a year of treatment 52% of patients receiving elafibranor had a significant reduction in cholestasis markers compared to 4% of patients receiving placebo. Initial changes were observed after the fourth week of treatment and were sustained for a year.
Although patients who received elafibranor experienced more adverse effects than those who received placebo, these were mild to moderate, and no patient had to stop treatment due to adverse effects. Common adverse effects included itching, weight gain, diarrhea, nausea, vomiting and fatigue.
“The results of the current trial showed that elafibranor may provide an effective, new second-line treatment for patients with primary biliary cholangitis,” the authors concluded.
