In patients with the rare, chronic cholestatic liver disease of primary biliary cholangitis, elafibranor significantly improved relevant biochemical indicators, researchers report. “The majority of patients enrolled in this trial were White, and although this feature aligns with the general epidemiology of the disease, racial minorities appeared to be underrepresented and ethnicity was not recorded,” write the authors. “The ongoing open-label extension and confirmatory phase 3 trial (ClinicalTrials.gov number, NCT06016842) are assessing additional data on the long-term safety of elafibranor and effects on clinical outcomes. The results of the current trial showed that elafibranor may provide an effective, new second-line treatment for patients with primary biliary cholangitis.”
The multinational, phase 3, double-blind ELATIVE trial included patients with primary biliary cholangitis who had had an inadequate response to or unacceptable side effects with ursodeoxycholic acid. Randomization to elafibranor 80 mg or placebo once daily produced these changes in a biochemical response (alkaline phosphatase level of <1.67 times the upper limit of the normal range, with a reduction of ≥15% from baseline, and normal total bilirubin levels) at week 52: “A total of 161 patients underwent randomization. A biochemical response (the primary end point) was observed in 51% of the patients (55 of 108) who received elafibranor and in 4% (2 of 53) who received placebo, for a difference of 47 percentage points (95% confidence interval [CI], 32 to 57; P <0.001). The alkaline phosphatase level normalized in 15% of the patients in the elafibranor group and in none of the patients in the placebo group at week 52 (difference, 15 percentage points; 95% CI, 6 to 23; P = 0.002). Among patients who had moderate-to-severe pruritus (44 patients in the elafibranor group and 22 in the placebo group), the least-squares mean change from baseline through week 52 on the WI-NRS did not differ significantly between the groups (−1.93 vs. −1.15; difference, −0.78; 95% CI, −1.99 to 0.42; P = 0.20). Adverse events that occurred more frequently with elafibranor than with placebo included abdominal pain, diarrhea, nausea, and vomiting.”