NDAORALCAPSULEPriority Review
Approved
Mar 2015
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
20
Data completeness
6/8 — Strong
Priority Review
CHOLBAM (cholic acid) by Mirum Pharmaceuticals is liver. Approved for bile acid synthesis defect, cerebrotendinous xanthomatosis, primary biliary cholangitis. First approved in 2015.
Drug data last refreshed Yesterday · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CHOLBAM (cholic acid) is an oral bile acid replacement therapy for rare genetic disorders affecting bile acid synthesis. It treats single enzyme defects (SEDs) and peroxisomal disorders like Zellweger spectrum disorder by restoring primary bile acid levels and preventing toxic accumulation of intermediate metabolites. The drug works by providing exogenous cholic acid to restore bile acid homeostasis and activate the farnesoid X receptor (FXR) pathway.
$0.004BPart D
PeakCHOLBAM is in peak commercial phase with modest Part D uptake of $4M in 2023, supporting a focused but stable commercial team structure.
Mechanism of Action
liver. In bile acid synthesis disorders due to SEDs in the biosynthetic pathway, and in PDs including Zellweger spectrum disorders, deficiency of primary bile acids leads to unregulated accumulation of intermediate bile acids and cholestasis. Bile acids facilitate fat digestion and absorption by…
Pharmacologic Class:
Bile Acid
Indications (3)
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.