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Bile acid Pregnancy: Women of childbearing potential should use an effective method of contraception. The use of oral contraceptives is not recommended in patients taking chenodeoxycholic acid. Pregnancy: Patients with CTX and high cholestanol have been shown to have adverse outcomes during pregnancy (two intrauterine deaths in a mother with CTX; two pregnancies resulting in premature infants with evidence of intrauterine growth retardation, reported in the literature). There are no or limited amount of data from the use of chenodeoxycholic acid in pregnant women. Studies in animals have shown reproductive toxicity. Chenodeoxycholic acid is not recommended during pregnancy and in women of childbearing potential not using contraception. Breast-feeding: it is unknown whether chenodeoxycholic acid/metabolites are excreted in human milk; a risk to the newborns/infants cannot be excluded; a decision must be made whether to discontinue breast-feeding or to discontinue/abstain from therapy. Fertility: anticipated to have no effects on fertility at therapeutic doses. (SPC 4.6)

Chenodeoxycholic acid

Brand names: Chenodeoxycholic acid Leadiant

Chenodeoxycholic acid is a naturally occurring primary bile acid used as replacement therapy in cerebrotendinous xanthomatosis, a rare inborn error of bile acid synthesis.

Adult dose

Dose: Cerebrotendinous xanthomatosis (CTX; inborn error of primary bile acid synthesis due to sterol-27 hydroxylase deficiency): starting dose 750 mg/day in three divided doses (250 mg hard capsules), provided that it is sufficient to normalise serum cholestanol and/or urine bile alcohols; if they remain elevated, increase the daily dose in 250 mg increments
Route: Oral (250 mg hard capsules)
Frequency: Three divided doses per day
Max: 1000 mg/day (adults)
Product: UK SPC via MHRA Products - CHENODEOXYCHOLIC ACID LEADIANT 250 MG HARD CAPSULES (PLGB449750001), Leadiant Biosciences Ltd. SPC 2: 'Each hard capsule contains 250 mg of chenodeoxycholic acid.' SPC 4.1 verbatim: 'Chenodeoxycholic acid is indicated for the treatment of inborn errors of primary bile acid synthesis due to sterol-27 hydroxylase deficiency (presenting as cerebrotendinous xanthomatosis (CTX)) in infants, children and adolescents 1 month to 18 years and adults.' CTX is the ONLY licensed indication; no gallstone-dissolution or other regimen is printed in this SPC. SPC 4.2 verbatim (adults): 'The starting dose in adults is 750 mg/day, in three divided doses provided that it is sufficient to normalise serum cholestanol and/or urine bile alcohols. The daily dose can be subsequently increased in 250 mg increments to a maximum of 1000 mg/ day if the serum cholestanol and/or urine bile alcohols remain elevated.' SPC 4.2 also: 'Treatment must be initiated and monitored by physicians experienced in the management of CTX or inborn errors of primary bile acid synthesis.' 'The lowest dose of chenodeoxycholic acid that effectively reduces the serum cholestanol and/or urine bile alcohols levels to within the normal range should be chosen.' 'In case of persistent lack of therapeutic response to chenodeoxycholic acid monotherapy, other treatment options should be considered.' Method of administration: oral use; can be taken with or without food; hard capsules should be taken whole with sufficient water at approximately the same time each day. Missed dose: take the next dose at the scheduled time; a double dose should not be taken to make up for the missed dose. Elderly (65 years and over): dose adjustment is not necessary. Overdose: concurrent elevation of liver enzymes above normal levels may indicate overdose.

Paediatric dose

Dose: CTX, 1 month to 18 years: starting dose 5 mg/kg/day in three divided doses
Route: Oral
Frequency: Three divided doses per day
Max: 15 mg/kg/day
SPC 4.2 Paediatric population (1 month - 18 years) verbatim: 'The starting dose in children is 5 mg/kg/day in three divided doses. Where the dose calculated is not a multiple of 250 mg, the nearest dose below the maximum of 15 mg/kg/day should be selected, provided that is sufficient to normalise serum cholestanol and/or urine bile alcohols.' These are per-DAY figures, not per-dose. Neonates less than one month of age: 'The safety and efficacy in neonates less than one month of age has not been established. Limited safety data are available' - no dose is given. For infants and children who cannot swallow capsules, the capsules may be carefully opened and the content added to sodium bicarbonate solution 8.4% (SPC refers to section 6.6, not retrieved here). SPC 4.8: in the only infant (diagnosed with CTX) enrolled in non-interventional studies, who started at 15 mg/kg/day, liver function tests rose within six weeks; they normalised on temporarily stopping treatment, and treatment was restarted at 5 mg/kg/day with no further complications. Verify against a children's formulary / the current SPC before use.

Dose adjustments

Renal

SPC 4.2 Renal impairment: 'No data are available for patients with renal impairment. However, these patients should be carefully monitored and the dose titrated individually.' No numeric renal dose adjustment is printed.

Hepatic

SPC 4.2 Hepatic impairment: 'No data are available for patients with hepatic impairment. However, these patients should be carefully monitored and the dose titrated individually.' No numeric hepatic dose adjustment is printed. Liver function should be monitored; concurrent elevation of liver enzymes above normal levels may indicate overdose.

Dose independently re-sourced from UK Summary of Product Characteristics (SPC) via MHRA Products — clinician-reviewed. Always confirm against the product SmPC and your local formulary before prescribing.

Contraindications

  • Hypersensitivity to the active substance or to any of the excipients listed in section 6.1 (SPC 4.3 - the only contraindication listed)

Side effects

  • Adverse reactions are 'generally mild to moderate in severity' (SPC 4.8)
  • Constipation - frequency not known
  • Transaminases increased - frequency not known
  • Jaundice - frequency not known
  • In two non-interventional studies, three adverse reactions in three of 63 patients, all non-serious (mild intermittent constipation in an adult and in a child; one hepatic reaction in a two-week-old infant with CTX, with multiple confounders)

Interactions

  • Colestipol or antacids containing aluminium hydroxide and/or smectite (aluminium oxide): do not give together - take either 2 hours before or after chenodeoxycholic acid (SPC 4.5)
  • Colestyramine: do not give together - take chenodeoxycholic acid either one hour before colestyramine or 4-6 hours after (SPC 4.5)
  • Ciclosporin and sirolimus: reduce chenodeoxycholic acid synthesis via CYP27A1; co-administration should be avoided; if considered necessary, closely monitor serum and urine bile alcohol levels and adjust the chenodeoxycholic acid dose (SPC 4.5)
  • Phenobarbital: increases HMG CoA reductase and counteracts one of the pharmacodynamic effects in CTX; if considered necessary, closely monitor serum and urine bile alcohol levels and adjust the dose (SPC 4.5)
  • Oral contraceptives: reduce the chenodeoxycholic acid pool size and may counteract its effectiveness in CTX - co-administration not recommended (SPC 4.5)
  • No interaction studies with concomitant medicines have been performed in patients with CTX (SPC 4.5)

Monitoring

  • Serum cholestanol and/or urine bile alcohols every 3 months during initiation and dose adjustment until metabolic control, then annually at a minimum (SPC 4.2)
  • Liver function - during initiation and then at least annually; concurrent elevation of liver enzymes above normal may indicate overdose (SPC 4.2)
  • Additional or more frequent investigations during periods of fast growth, concomitant disease and pregnancy (SPC 4.2)
  • Patients with renal or hepatic impairment: careful monitoring with individual dose titration (SPC 4.2)

Clinical monograph

How it works

By replacing the deficient bile acid it restores feedback inhibition of cholesterol 7-alpha-hydroxylase, reducing the abnormal production and accumulation of cholestanol and bile alcohols that characterise the disorder.

Prescribing in practice

  • Liver function must be monitored because bile acids can cause hepatotoxicity, and treatment should be reviewed if there is evidence of significant liver injury.
  • It is a treatment for the rare metabolic disorder cerebrotendinous xanthomatosis and should be initiated and supervised by specialists experienced in its management.
  • Concurrent bile-acid sequestrants and certain aluminium-containing antacids can bind bile acid and reduce its effect, so dosing should be separated.

Monitoring

Monitor liver function regularly during treatment, together with relevant biochemical markers of the underlying metabolic disorder.

Counselling the patient

  • This is long-term replacement therapy for a rare inherited condition; take it consistently.
  • Attend for the blood tests used to check your liver and treatment response.
  • Separate it from any cholesterol-binding or antacid medicines you take.

Evidence & guidelines

Bile acid replacement is the established treatment for cerebrotendinous xanthomatosis, supported by long-term observational data showing biochemical and clinical improvement.

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).

Related

Curated clinical cross-links plus same-class fallbacks.

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