norursodeoxycholic acid

norursodeoxycholic acid (norUDCA) is a synthetic, side-chain-shortened derivative of ursodeoxycholic acid (UDCA), classified as a small molecule bile acid. It is under clinical investigation primarily for the treatment of primary sclerosing cholangitis (PSC), an immune-mediated cholestatic liver disease with limited effective therapies. norUDCA exhibits potent anti-cholestatic, anti-inflammatory, and anti-fibrotic properties in preclinical models and has demonstrated significant reductions in serum alkaline phosphatase levels in phase II/III clinical trials for PSC. Its mechanism involves resistance to amidation, enabling cholehepatic shunting and hepatic enrichment, which facilitates direct effects on both parenchymal and non-parenchymal liver cells—counteracting cholestasis, steatosis, inflammation, fibrosis, inhibiting hepatocellular proliferation, promoting autophagy, and modulating CD8+ T cell-driven immunopathology. The drug has orphan designation status in the EU for PSC[1][2][3][5][6].

Brand names

NUC (Dr Falk Pharma)

Other names

24-Nor Ursodeoxycholic AcidNORUCHOLIC ACID99697-24-2