NorUDCA FDA Approval Status
Norursodeoxycholic acid (norUDCA) is NOT approved by the US Food and Drug Administration (FDA) for any indication and remains an investigational agent currently undergoing clinical trials. 1, 2
Current Regulatory Status
NorUDCA has no large phase III trials completed for any indication, and no published histological fibrosis endpoint data exists for norUDCA in human trials 2
The drug is being investigated as a novel therapeutic agent for primary sclerosing cholangitis (PSC), with phase II trials showing dose-dependent reductions in alkaline phosphatase levels (up to -26% at 1,500mg/day) with an excellent safety profile 1, 3
The European Association for the Study of the Liver identifies norUDCA as a distinct "novel bile acid-based therapeutic option" separate from standard UDCA, alongside FXR agonists and other emerging therapies 1
Distinction from UDCA
NorUDCA is structurally distinct from ursodeoxycholic acid (UDCA) - it is a side-chain-shortened C23 derivative of UDCA, not a conjugated form 1, 4
The resistance to conjugation gives norUDCA distinct therapeutic properties, including anti-cholestatic, anti-inflammatory, anti-fibrotic, and antiproliferative effects that differ from standard UDCA 1, 5
Unlike UDCA (which IS FDA-approved for primary biliary cirrhosis), norUDCA remains investigational 2
Clinical Trial Evidence
A randomized controlled trial including 38 centers from 12 European countries evaluated three doses of oral norUDCA (500mg/day, 1,000mg/day, or 1,500mg/day) in 161 PSC patients over 12 weeks 3
The safety profile was excellent and comparable to placebo, with no difference in reported pruritus between treatment and placebo groups 3
These phase II results justify a phase III trial, but no FDA approval has been granted 3
Important Caveat
The FDA drug label references 6 in the evidence appear to be for a different product (likely an iron-containing supplement based on the overdose warning), not for norUDCA specifically, as norUDCA has no approved FDA labeling.