Bile acid replacement Pregnancy: No studies in pregnant women and no animal reproduction studies have been conducted. Limited published case reports of pregnancies in women taking cholic acid for 3-beta-HSD deficiency resulted in healthy infants, but are insufficient to establish presence or absence of drug-associated risk. A pregnancy surveillance programme monitors outcomes.

Cholic acid

Brand names: Orphacol, Kolbam

Cholic acid is a primary bile acid used as long-term replacement therapy in patients with inborn errors of primary bile acid synthesis.

Auto-extracted from the source labelling — not yet independently clinician-verified. These values were distilled from the UK SPC (or the US label where noted) but have not had a clinician sign-off. Confirm against the current SmPC before prescribing.

Adult dose

Dose: 10 to 15 mg/kg

Route: Oral (capsules, taken with food)

Frequency: Once daily or in two divided doses

Max: No maximum dose is stated in the source label

Weight-based in adults as well as children. Indication in the source label (CHOLBAM): bile acid synthesis disorders due to single enzyme defects (SEDs), and adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders with liver disease, steatorrhoea or complications of decreased fat-soluble vitamin absorption. In patients with concomitant familial hypertriglyceridaemia the recommended dosage is 11 to 17 mg/kg once daily or in two divided doses (poor intestinal absorption; about a 10% increase to account for malabsorption), adjusted on clinical response. Administer the lowest dose that effectively maintains liver function. Treatment should be initiated and monitored by an experienced hepatologist or paediatric gastroenterologist. Monitor AST, ALT, GGT, alkaline phosphatase, bilirubin and INR monthly for the first 3 months, every 3 months for the next 9 months, every 6 months for the next three years, then annually; monitor more frequently during rapid growth, concomitant disease and pregnancy. Discontinue if liver function does not improve within 3 months, if complete biliary obstruction develops, or if there are persistent indicators of worsening liver function or cholestasis; consider restarting at a lower dose when parameters return to baseline. Concurrent elevations of serum GGT and ALT may indicate overdose. Administration: take with food; take at least 1 hour before, or 4 to 6 hours after, a bile acid binding resin or aluminium-based antacid; do not crush or chew the capsules — if unable to swallow, open the capsule and mix the contents with 15-30 mL of infant formula, expressed breast milk or soft food and give immediately. The label gives capsule-count tables for 10 mg/kg/day and 15 mg/kg/day using 50 mg and 250 mg capsules. US labelling — no UK SPC was available in this bundle.

Paediatric dose

Route: Oral

Frequency: Once daily or in two divided doses

Max: No maximum dose is stated in the source label

The source label states the same weight-based regimen for paediatric patients and adults: 10 to 15 mg/kg/day (11 to 17 mg/kg/day with concomitant familial hypertriglyceridaemia). A single dosePerKg value has deliberately not been asserted because the label states a range, not a point dose. Safety and effectiveness established in paediatric patients 3 weeks of age and older. US labelling — verify the regimen and any UK/EU product differences against a children's formulary and the specialist centre's protocol before use.

Dose auto-extracted from US FDA prescribing information (openFDA / DailyMed) — cross-check; US labelling may differ from UK — not yet clinician-verified. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

None stated (US label §4: 'None')

Side effects

Diarrhoea
Reflux oesophagitis
Nausea and abdominal pain
Jaundice and exacerbation of liver impairment (severe hepatotoxicity reported post-marketing in patients with cirrhosis)
Malaise, skin lesion, peripheral neuropathy

Interactions

Bile salt efflux pump (BSEP) inhibitors, e.g. ciclosporin — avoid concomitant use; if necessary, monitor serum transaminases and bilirubin
Bile acid binding resins (colestyramine, colestipol, colesevelam) adsorb bile acids and may reduce efficacy — give cholic acid at least 1 hour before or 4 to 6 hours after
Aluminium-based antacids — separate dosing as above

Clinical monograph

How it works

Replacing the missing bile acid restores negative feedback on bile acid synthesis, reducing the production of hepatotoxic intermediate metabolites while supporting bile flow and fat-soluble vitamin absorption.

Prescribing in practice

Liver function should be monitored throughout treatment, as worsening liver biochemistry may indicate an inappropriate dose or progression requiring review.
It is indicated for rare inborn errors of bile acid synthesis and should be managed by clinicians experienced in these metabolic disorders.
Bile-acid sequestrants and some antacids reduce its absorption, so administration should be appropriately separated.

Monitoring

Monitor liver function and serum and urinary bile acid metabolites to guide dosing and confirm therapeutic response.

Counselling the patient

This replaces a bile acid your body cannot make properly and is taken long term.
Keep your appointments for the blood and urine tests that guide your dose.
Leave a gap between this and any cholesterol-lowering resin or antacid.

Evidence & guidelines

Cholic acid replacement is the recognised treatment for inborn errors of primary bile acid synthesis and is supported by long-term observational outcome data.

Reference: Drug verified in RxNorm (NLM); confirm dosing against the manufacturer SPC (eMC). Verify against your local formulary and current prescribing references before prescribing. The structured dose values shown have been reviewed by a clinician. Monograph status: clinician-reviewed (2026-07-04).

Curated clinical cross-links plus same-class fallbacks.

Calculators

Pathways