- The biochemical efficacy of bezafibrate, an agonist of peroxisome proliferator-activated receptors (PPARs), plus ursodeoxycholic acid (UA) is superior to placebo plus UA in patients with primary biliary cholangitis who have inadequate response to UA.
Why this matters
- 40% of patients with primary biliary cholangitis have inadequate response to UA.
- This study represents the first treatment advance in 2 decades.
- Randomized, double-blind, multicenter, placebo-controlled 24-month phase 3 trial of 100 patients with inadequate response to UA, randomly assigned to add-on bezafibrate (400 mg daily) or placebo.
- Primary outcome was complete biochemical response, defined as normal levels of total bilirubin, alkaline phosphatase, aminotransferases, albumin, and normal prothrombin index.
- Funding: Ministry of Health, France; Arrow Generiques, France.
- The bezafibrate group had higher rates of complete biochemical response than the placebo group (31% vs 0%, respectively; P<.001>
- The bezafibrate group was more likely to have normal levels of alkaline phosphatase (67% vs 2%; difference of 65 percentage points; 95% CI, 47-79).
- The bezafibrate group had improvements in pruritus, fatigue, noninvasive measures of liver fibrosis similar in extent to the primary outcome (P values not given).
- Safety: the bezafibrate group had slightly higher levels of creatinine.
- Clinical outcomes less studied.