Odevixibat (A4250) is an effective, selective and orally active ileal bile acid transporter inhibitor. Odevixibat has the potential for the treatment of primary biliary cirrhosis. Odevixibat reduces cholestatic liver and bile duct injury in mice model.

Odevixibat obviously decreases bile flow and biliary BA output, which correlates with reduced bsep transcription, while Ntcp and Cyp7a1 are induced. Odevixibat (0.01% (w/w) in chow diet; 4 weeks) improves sclerosing cholangitis and significantly decreases serum alanine aminotransferase, alkaline phosphatase and BAs levels, hepatic expression of pro-inflammatory and pro-fibrogenic genes and bile duct proliferation in Mdr2-/- mice [1].