Last reviewed · How we verify
Cholbam
At a glance
| Generic name | Cholbam |
|---|---|
| Sponsor | Mirum Pharmaceuticals, Inc. |
| Target | G-protein coupled bile acid receptor 1, Bile acid receptor |
| Modality | Small molecule |
| Therapeutic area | Metabolic |
| Phase | FDA-approved |
Approved indications
- 3-Beta-hydroxy-delta-5-C27-steroid dehydrogenase deficiency
- Calculus in biliary tract
- Cholestanol storage disease
- Constipation
- Delta-4-3-oxosteroid-5-beta-reductase deficiency
- Disorder of biliary tract
- Inborn error of bile acid metabolism
- Incomplete passage of stool
Common side effects
- Diarrhea
- Reflux Esophagitis
- Malaise
- Jaundice
- Skin Lesion
- Nausea
- Abdominal Pain
- Intestinal Polyp
- Urinary Tract Infection
- Peripheral Neuropathy
Serious adverse events
- Death (SED patients ≤1 year)
- Death (PD patients 7 months-2.5 years)
- Worsening Liver Impairment
- Symptomatic Cholelithiasis requiring Cholecystectomy
Key clinical trials
- Quantitative Liver Function Tests Using Cholates (PHASE2)
- Cholic Acid for Hepatic Steatosis in Lipodystrophy (PHASE2)
- Efficacy of Lactobacillus Paracasei LC19 on Type 2 Diabetes (NA)
- Smith-Lemli-Opitz Syndrome and Cholic Acid (PHASE1, PHASE2)
- Efficacy of To Be Marketed (TBM) Cholic Acid Capsules Used to Treat Children With Inborn Errors of Bile Acid Synthesis (PHASE3)
- Open Label, Continuation Study of Cholic Acid in Subjects With Inborn Errors of Bile Acid Synthesis (PHASE3)
- Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid (PHASE3)
- The REPLACE Registry for Cholbam® (Cholic Acid)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Cholbam CI brief — competitive landscape report
- Cholbam updates RSS · CI watch RSS
- Mirum Pharmaceuticals, Inc. portfolio CI