NDAORALCAPSULEPriority Review
Approved
Mar 2015
Lifecycle
Peak
Competitive Pressure
30/100
Clinical Trials
20
Data completeness
5/8 — Partial
Priority Review
CHOLBAM (cholic acid) by Mirum Pharmaceuticals is liver. First approved in 2015.
Drug data last refreshed 5h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CHOLBAM (cholic acid) is an oral bile acid replacement therapy indicated for rare genetic disorders affecting bile acid synthesis, including cerebrotendinous xanthomatosis (CTX) and Zellweger spectrum disorders. It works by replacing deficient primary bile acids and providing feedback inhibition of abnormal bile acid synthesis through farnesoid X receptor (FXR) activation. The drug restores bile acid homeostasis and prevents accumulation of toxic intermediate metabolites.
$0.004BPart D
PeakEstablished niche product with stable commercial footprint across rare disease specialists; small, specialized sales organization focused on metabolic disorder centers.
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
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
Comparative Evaluation of Oral Ursodeoxycholic Acid in Reducing Bilirubin Levels Among Patients With Acute Viral Hepatitis
Started Apr 2026
88 enrolled
Hepatitis AHepatitis E Virus Infection
Study with Norucholic Acid Tablets in Patients with Primary Sclerosing Cholangitis (PSC)
Started Mar 2025
120 enrolled
Primary Sclerosing Cholangitis
A Study of Elafibranor in Adults With Primary Biliary Cholangitis and Inadequate Response or Intolerance to Ursodeoxycholic Acid.
Started Jul 2024
69 enrolled
Primary Biliary Cholangitis
A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
Started May 2024
0Cerebrotendinous Xanthomatoses
Linerixibat and Obeticholic Acid Drug Interaction Study in Healthy Adult Participants
Started Nov 2021
52 enrolled
Pruritus
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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.