Small molecule · Activator of farnesoid X receptor (NR1H4) · developed by Leadiant Biosci
CDCA (chenodeoxycholic acid) is an endogenous primary bile acid that replaces deficient levels in cerebrotendinous xanthomatosis caused by sterol 27-hydroxylase deficiency. It restores farnesoid X receptor activation and suppresses atypical bile acid synthesis, reducing cholestanol and pentol accumulation.
Summary written with AI from the sources listed at the end of this page.
From the indications section of the FDA label (version of Dec 16, 2025). Read the full label for the exact wording, limitations and safety information.
| Trial | Phase | Condition | Status | Sponsor |
|---|---|---|---|---|
| NCT04270682 | Phase 3 | CTX | Completed | MIRM |
Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.
CTX
Updated Oct 3, 2026.