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Cholic Acids

Mirum Pharmaceuticals, Inc. · Phase 3 active Small molecule

Cholic acid is a primary bile acid that restores bile acid-dependent signaling pathways to improve cholestasis and fat malabsorption in patients with bile acid synthesis disorders.

Cholic acid is a primary bile acid that restores bile acid-dependent signaling pathways to improve cholestasis and fat malabsorption in patients with bile acid synthesis disorders. Used for Cerebrotendinous xanthomatosis (CTX), Progressive familial intrahepatic cholestasis (PFIC), Bile acid synthesis disorders.

At a glance

Generic nameCholic Acids
Also known asCholic, Cholic Acid, Cholic Acid Capsules
SponsorMirum Pharmaceuticals, Inc.
Drug classBile acid replacement therapy
TargetFarnesoid X receptor (FXR) and TGR5 signaling pathways
ModalitySmall molecule
Therapeutic areaGastroenterology / Metabolic Disorders
PhasePhase 3

Mechanism of action

Cholic acid functions as a replacement therapy for patients with deficient endogenous bile acid synthesis, particularly in cerebrotendinous xanthomatosis (CTX) and other inborn errors of bile acid metabolism. By supplementing the deficient primary bile acid pool, it restores normal enterohepatic circulation, improves fat-soluble vitamin absorption, and reduces accumulation of toxic bile acid precursors. This mechanism addresses the underlying metabolic defect rather than treating symptoms alone.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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