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CDCA Weight-Based Dose TID

Mirum Pharmaceuticals, Inc. · Phase 3 active Small molecule

CDCA (chenodeoxycholic acid) is a bile acid that activates farnesoid X receptor (FXR) and TGR5 signaling to reduce bile acid synthesis and improve cholestasis.

CDCA (chenodeoxycholic acid) is a bile acid that activates farnesoid X receptor (FXR) and TGR5 signaling to reduce bile acid synthesis and improve cholestasis. Used for Progressive familial intrahepatic cholestasis (PFIC) type 1, Progressive familial intrahepatic cholestasis (PFIC) type 2.

At a glance

Generic nameCDCA Weight-Based Dose TID
SponsorMirum Pharmaceuticals, Inc.
Drug classBile acid receptor agonist
TargetFXR (farnesoid X receptor), TGR5
ModalitySmall molecule
Therapeutic areaHepatology / Gastroenterology
PhasePhase 3

Mechanism of action

CDCA is an endogenous bile acid that acts as a ligand for nuclear farnesoid X receptor (FXR) and G-protein coupled receptor TGR5. Activation of FXR suppresses CYP7A1, the rate-limiting enzyme in bile acid synthesis, thereby reducing hepatic bile acid production and accumulation. This mechanism helps alleviate cholestasis and associated pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) and other cholestatic liver diseases.

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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