Norursodeoxycholic Acid for Liver Fibrosis Treatment

Current Evidence Status

• Nor-UDCA has demonstrated anti-fibrotic properties in preclinical models and early human trials, but currently lacks definitive evidence for fibrosis improvement in clinical practice, with only preliminary data showing biochemical improvements in metabolic liver disease 1
• Nor-UDCA has shown anti-cholestatic, anti-inflammatory, and anti-fibrotic properties in preclinical models, distinguishing it from standard UDCA 1, 2
• Initial results from MASH trials show improvement in ALT and liver steatosis, but no published data yet on fibrosis endpoints 1, 2

Comparison to Standard UDCA

• Standard UDCA has no proof of histological efficacy in MASH despite biochemical improvements 1, 2
• UDCA is not recommended for preventing advanced liver disease in cystic fibrosis, as studies show no difference in incidence of advanced liver disease 3, 4, 5, 6, 7

Clinical Application Considerations

• No large phase III trials have been completed for nor-UDCA in any liver disease with fibrosis as a primary endpoint 1
• The existing human data is limited to biochemical markers and imaging surrogates rather than histological fibrosis assessment 1, 2