Treatment with elafibranor may improve bile flow in patients with primary biliary cholangitis (PBC), according to results of a phase 3 trial recently published in The New England Journal of Medicine.
Elafibranor was developed by Ipsen and Genfit, and both companies funded the trial. The aim of this study was to investigate whether elafibranor, an oral experimental therapy for the treatment of PBC, may provide any benefit to patients. The trial enrolled 161 patients with PBC who had a poor response or suffered serious side effects when treated with ursodeoxycholic acid, the current standard of care.
Patients were randomly assigned to groups receiving 80 mg of elafibranor or placebo (inactive substance), once per day. The researchers evaluated the effectiveness of the drug through relevant biochemical indicators – the level of alkaline phosphatase and bilirubin.
Read more about PBC therapies
Alkaline phosphatase is a molecule primarily found in the liver and bones. When liver cells get damaged, it is released into the bloodstream. Bilirubin passes through the liver and bile canals on its way out of the body and is a useful indicator of the gallbladder and biliary tract health. High alkaline phosphatase and bilirubin levels may indicate liver disease or bile blockage.
In this study, a positive biochemical response to elafibranor was defined as the level of alkaline phosphatase lower than 1.67 times the upper limit of the normal range, with a reduction of at least 15% from the start of the treatment, and normal bilirubin levels, at week 52.
The researchers also monitored the normalization of the alkaline phosphatase level and the change in the intensity of itchiness.
According to the results, 51% of patients who received elafibranor recorded a positive biochemical response to treatment, compared with 4% of those who received placebo. The level of alkaline phosphatase normalized in 15% of the patients who received elafibranor and in none of the patients who were treated with placebo.
The itchiness intensity remained similar in both groups. Patients treated with elafibranor experienced stomach pain, diarrhea, nausea, and vomiting more often than those treated with placebo.
