CHENODIOL · LGM PHARMA
Chenodiol is a bile acid used for adults living with cerebrotendinous xanthomatosis (CTX). It helps patients with this condition by replacing the naturally occurring chenodeoxycholic acid that their bodies are unable to produce sufficiently. By restoring these levels, the drug addresses the metabolic disruptions caused by a deficiency in the sterol 27-hydroxylase enzyme.
How CHENODIOL Works
This drug works by replacing deficient levels of endogenous chenodeoxycholic acid, which restores the activation of the farnesoid X receptor (FXR). This activation downregulates the CYP7A1 enzyme, resulting in the suppression and reduction of atypical bile acids and alcohols such as cholestanol and 23S-pentol.
Details
- Status
- Prescription
- First Approved
- 1983-07-28
- Routes
- ORAL
- Dosage Forms
- TABLET
CHENODIOL Approval History
What CHENODIOL Treats
1 indicationsCHENODIOL is approved for 1 conditions since its original approval in 2009. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Cerebrotendinous Xanthomatosis
CHENODIOL Boxed Warning
SPECIAL NOTE Because of the potential hepatoxicity of chenodiol, poor response rate in some subgroups of chenodiol treated patients, and an increased rate of a need for cholecystectomy in other chenodiol treated subgroups, chenodiol is not an appropriate treatment for many patients with gallstones. Chenodiol should be reserved for carefully selected patients and treatment must be accompanied by systematic monitoring for liver function alterations. Aspects of patient selection, response rates and...
SPECIAL NOTE Because of the potential hepatoxicity of chenodiol, poor response rate in some subgroups of chenodiol treated patients, and an increased rate of a need for cholecystectomy in other chenodiol treated subgroups, chenodiol is not an appropriate treatment for many patients with gallstones. Chenodiol should be reserved for carefully selected patients and treatment must be accompanied by systematic monitoring for liver function alterations. Aspects of patient selection, response rates and risks versus benefits are given in the insert.
CHENODIOL Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
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Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
CHENODIOL FDA Label Details
Indications & Usage
Chenodiol is indicated for patients with radiolucent stones in well-opacifying gallbladders, in whom selective surgery would be undertaken except for the presence of increased surgical risk due to systemic disease or age. The likelihood of successful dissolution is far greater if the stones are floatable or small. For patients with nonfloatable stones, dissolution is less likely and added weight should be given to the risk that more emergent surgery might result form a delay due to unsuccessful treatment. Safety of use beyond 24 months is not established. Chenodiol will not dissolve calcified ...
SPECIAL NOTE Because of the potential hepatoxicity of chenodiol, poor response rate in some subgroups of chenodiol treated patients, and an increased rate of a need for cholecystectomy in other chenodiol treated subgroups, chenodiol is not an appropriate treatment for many patients with gallstones. ...
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CTEXLI
Full clinical data, patents, trials, and competitive landscape for chenodiol.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.