CHOLBAM (cholic acid) · MIRUM
Cholbam (cholic acid) is a primary bile acid indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). It is also indicated as adjunctive treatment for peroxisomal disorders (PDs), including Zellweger spectrum disorders, in patients exhibiting manifestations of liver disease, steatorrhea, or complications from fat-soluble vitamin malabsorption. Limitations of Use: The safety and effectiveness of Cholbam on extrahepatic manifestations of SEDs or PDs have not been established.
How CHOLBAM Works
Cholic acid is an endogenous primary bile acid that addresses deficiencies resulting from biosynthetic pathway defects. In SEDs and PDs, the lack of primary bile acids leads to the accumulation of toxic intermediate bile acids and cholestasis. Cholic acid acts as a ligand for the farnesoid X receptor (FXR), which provides physiologic feedback inhibition of bile acid synthesis, thereby reducing the production of harmful intermediates. Furthermore, it restores the formation of mixed micelles in the intestine, facilitating the digestion and absorption of dietary fats and fat-soluble vitamins.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2015-03-17
- Routes
- ORAL
- Dosage Forms
- CAPSULE
CHOLBAM Approval History
What CHOLBAM Treats
5 indicationsCHOLBAM is approved for 5 conditions since its original approval in 2015. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Bile Acid Synthesis Disorders
- Peroxisomal Disorders
- Zellweger Spectrum Disorders
- Liver Disease
- Steatorrhea
CHOLBAM 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?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
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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Other CDER-designated drugs from the same sponsor (2016–2025).
Clinical Trial Registry
3 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03720990 results posted | 0464-18-FB | Ph 1, Ph 2 | completed | Smith-Lemli-Opitz Syndrome and Cholic Acid |
| NCT01115582 results posted | CAC-001-01 | Ph 3 | completed | Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis |
| NCT01438411 results posted | CAC-002-01 | Ph 3 | completed | Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis |
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.
CHOLBAM FDA Label Details
Indications & Usage
FDA Label (PDF)CHOLBAM is a bile acid indicated for: • Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). • Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption. Limitations of use: The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to SEDs or PDs including Zellweger spectrum disorders have not been established. . 1.1 Bile Acid Synthesis Disorders Due to Si...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment