CHOLBAM
Clinical safety rating: caution
Comprehensive clinical and safety monograph for CHOLBAM (CHOLBAM).
Cholic acid is a primary bile acid that acts as a peroxisome proliferator-activated receptor alpha (PPARα) agonist, reducing the synthesis of bile acids from cholesterol via feedback inhibition of the rate-limiting enzyme cholesterol 7α-hydroxylase (CYP7A1). It also improves bile flow and reduces the accumulation of toxic bile acid intermediates.
| Metabolism | Cholic acid is conjugated with glycine or taurine to form conjugated bile acids. It undergoes enterohepatic recirculation and is deconjugated by intestinal bacteria. Minor metabolism via hydroxylation and oxidation. Excreted primarily in feces. |
| Excretion | Primarily biliary (fecal) excretion as conjugates; less than 5% renal excretion as unchanged drug and metabolites. |
| Half-life | Terminal elimination half-life approximately 3.5-7 hours in patients with primary bile acid synthesis disorders; may be prolonged in severe hepatic impairment. |
| Protein binding | Greater than 90% bound to plasma proteins, primarily to albumin. |
| Volume of Distribution | Approximately 0.2-0.4 L/kg, suggesting distribution primarily in plasma and interstitial fluid. |
| Bioavailability | Oral: approximately 50-70% after absorption, but extensive first-pass hepatic extraction reduces systemic availability. |
| Onset of Action | Oral: reduction of serum bile acid levels and improvement in clinical symptoms observed within days to weeks of initiating therapy. |
| Duration of Action | Duration of therapeutic effect persists with regular dosing; clinical effects maintained as long as therapy continues, with wear-off within days to weeks after discontinuation. |
10-15 mg/kg orally once daily; maximum daily dose 500 mg.
| Dosage form | CAPSULE |
| Renal impairment | No specific dose adjustment guidelines; use with caution in severe renal impairment (GFR <30 mL/min). |
| Liver impairment | Contraindicated in Child-Pugh class B or C hepatic impairment; no adjustment for Child-Pugh class A (mild). |
| Pediatric use | 10-15 mg/kg orally once daily; maximum 500 mg/day. Safety and efficacy in neonates not established. |
| Geriatric use | No specific dose adjustments; monitor renal and hepatic function due to age-related decline. |
| 1st trimester | Consult provider |
| 2nd trimester | Consult provider |
| 3rd trimester | Consult provider |
Clinical note
Comprehensive clinical and safety monograph for CHOLBAM (CHOLBAM).
| Breastfeeding | It is unknown if cholic acid is excreted in human milk. No data on M/P ratio. Due to the lack of safety data, caution is advised; the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CHOLBAM. |
| Teratogenic Risk | CHOLBAM (cholic acid) is a bile acid used for bile acid synthesis disorders. In animal studies, no teratogenic effects were observed at clinically relevant doses. Human data are limited, but bile acids are critical for fetal development; deficiency may cause fetal harm. Use during pregnancy only if clearly needed. The risk is likely low, but potential for fetal harm cannot be excluded. |
■ FDA Black Box Warning
None
| Serious Effects |
["Hypersensitivity to cholic acid or any component of the formulation.","Patients with complete biliary obstruction."]
| Precautions | ["May exacerbate liver disease in patients with certain bile acid synthesis defects not responsive to therapy.","Risk of drug-induced liver injury; monitor liver function tests.","Potential for diarrhea and other gastrointestinal disturbances.","May interfere with the absorption of fat-soluble vitamins (A, D, E, K); monitor vitamin levels."] |
| Food/Dietary | Cholbam should be taken with food to improve absorption and reduce gastrointestinal side effects. Avoid high-fat meals that may alter bile acid metabolism; maintain consistent dietary fat intake. No specific food restrictions are mandated, but patients should follow a balanced diet as recommended by their healthcare provider. |
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| Fetal Monitoring | Monitor liver function tests (bilirubin, AST, ALT, GGT), serum bile acids, and prothrombin time (INR) in pregnant women receiving CHOLBAM. Fetal monitoring should include ultrasound for growth and amniotic fluid volume, and non-stress testing as clinically indicated, especially in third trimester. |
| Fertility Effects | No formal studies on fertility effects. In animal studies, no adverse effects on fertility were noted at clinically relevant doses. Human data are insufficient to assess impact on fertility. |
| Clinical Pearls | Cholbam (cholic acid) is used for bile acid synthesis disorders due to single enzyme defects, and for peroxisomal disorders such as Zellweger spectrum disorders. Monitor liver function tests and bile acid levels regularly. Dosage must be adjusted based on body weight and clinical response. May cause gastrointestinal adverse effects; administer with food to reduce GI upset. Safety in pregnancy not established. |
| Patient Advice | Take Cholbam exactly as prescribed, usually twice daily with food. · Do not stop or change dose without consulting your doctor. · Report any new or worsening symptoms such as jaundice, abdominal pain, or diarrhea. · Regular blood tests are needed to monitor liver function and bile acid levels. · Inform your doctor if you are pregnant, planning to become pregnant, or breastfeeding. · Store at room temperature away from moisture and heat. |