Kris V Kowdley, Christopher L Bowlus, Cynthia Levy, Ulus S Akarca, Mario Reis Alvares-da-Silva, Pietro Andreone, Marco Arrese, Christophe Corpechot, Sven M Francque, Michael A Heneghan, Pietro Invernizzi, David Jones, Frederik C Kruger, Eric Lawitz, Marlyn J Mayo, Mitchell L Shiffman, Mark G Swain, José Miguel Valera, Victor Vargas, John M Vierling, Alejandra Villamil, Carol Addy, Julie Dietrich, Jean-Michel Germain, Sarah Mazain, Dragutin Rafailovic, Bachirou Taddé, Benjamin Miller, Jianfen Shu, Claudia O Zein, Jörn M Schattenberg
BACKGROUND: Primary biliary cholangitis is a rare, chronic cholestatic liver disease characterized by the destruction of interlobular bile ducts, leading to cholestasis and liver fibrosis. Whether elafibranor, an oral, dual peroxisome proliferator-activated receptor (PPAR) α and δ agonist, may have benefit as a treatment for primary biliary cholangitis is unknown. METHODS: In this multinational, phase 3, double-blind, placebo-controlled trial, we randomly assigned (in a 2:1 ratio) patients with primary biliary cholangitis who had had an inadequate response to or unacceptable side effects with ursodeoxycholic acid to receive once-daily elafibranor, at a dose of 80 mg, or placebo...
November 13, 2023: New England Journal of Medicine