pharmacopeia

Cholic Acid

Bile acids and derivativesBile AcidUS-FDASourced from openFDA
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Mechanism of action

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Cholic acid is a primary bile acid synthesized from cholesterol in the 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.

Indications

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  • CHOLBAM is a bile acid indicated for: • Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). ( 1.1 ) • Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption.

Contraindications

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  • None.contraindicated

Dosage & administration

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• The recommended dosage is 10 to 15 mg/kg once daily or in two divided doses, in pediatric patients and adults. See prescribing information for weight-based dosing tables. ( 2.1 ) • The recommended dosage in patients with concomitant familial hypertriglyceridemia is 11 to 17 mg/kg once daily or in two divided doses and is adjusted based on clinical response. ( 2.1 ) • Monitor AST, ALT, GGT, alkaline phosphatase, bilirubin and INR every month for the first 3 months, every 3 months for the next 9 months, every 6 months during the next three years and annually thereafter. Administer the lowest dose that effectively maintains liver function. ( 2.2 ) • Discontinue CHOLBAM if liver function does not improve within 3 months of starting treatment, if complete biliary obstruction develops, or if there are persistent clinical or laboratory indicators of worsening liver function or cholestasis; continue to monitor liver function and consider restarting at a lower dose when parameters return to baseline. ( 2.2 , 5.1 ) Administration Instructions: • Take with food. ( 2.3 ) • Do not crush or chew the capsules. For patients unable to swallow the capsules, the capsules can be opened and the contents mixed with drink/food ( 2.3 ) 2.1. Dosage Regimen for Bile Acid Synthesis Disorders Due to SEDs and PDs Including Zellweger Spectrum Disorders The recommended dosage of CHOLBAM is 10 to 15 mg/kg administered orally once daily or in two divided doses, in pediatric patients and adults.

Warnings & precautions

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Exacerbation of Liver Impairment: Monitor liver function. Discontinue CHOLBAM if liver function worsens while on treatment. ( 5.1 ) 5.1 Exacerbation of Liver Impairment In clinical trials, evidence of liver impairment was present before treatment with CHOLBAM in approximately 86% (44/51) of patients with bile acid synthesis disorders due to SEDs and in approximately 50% (14/28) of patients with PDs including Zellweger spectrum disorders. Five of the patients (3 SED and 2 PD) with liver impairment at baseline experienced worsening serum transaminases, elevated bilirubin values, or worsening cholestasis on liver biopsy following treatment. Five additional patients (2 SED and 3 PD) who did not have baseline cholestasis experienced exacerbation of their liver disease while on treatment. In patients with cirrhosis, cases of severe hepatotoxicity have also been observed following postmarket use of CHOLBAM. Exacerbation of liver impairment by CHOLBAM in these patients cannot be ruled out. Six patients with SEDs underwent liver transplant, including four patients diagnosed with AKR1D1 deficiency, one with 3β-HSD deficiency, and one with CYP7A1 deficiency. Concurrent elevations of serum GGT and ALT may indicate CHOLBAM overdose [see Dosage and Administration ( 2.2 ) and Overdosage ( 10 )] . Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis [see Dosage and Administration ( 2.2 )].

Adverse reactions

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The following clinically significant adverse reaction is described elsewhere in the labeling: • Exacerbation of Liver Impairment [see Warnings and Precautions ( 5.1 )] Most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Mirum Pharmaceuticals at 1-855-MRM-4YOU or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Clinical safety experience with CHOLBAM consists of: • Trial 1: a non-randomized, open-label, single-arm trial of 50 patients with bile acid synthesis disorders due to SEDs and 29 patients with PDs including Zellweger spectrum disorders. Safety data are available over the 18 years of the trial. • Trial 2: an extension trial of 12 new patients (10 SED and 2 PD) along with 31 (21 SED and 10 PD) patients who rolled over from Trial 1. Safety data are available for 3 years and 11 months of treatment. Adverse events were not collected systematically in either of these trials. Most patients received an oral dose of 10 to 15 mg/kg/day of CHOLBAM.

Use in specific populations

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8.1 Pregnancy Pregnancy Surveillance Program There is a pregnancy surveillance program that monitors pregnancy outcomes in women exposed to CHOLBAM during pregnancy. Women who become pregnant during CHOLBAM treatment are encouraged to enroll. Patients or their health care provider should call Mirum Medical Information 1-855-676-4968. Risk Summary No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM. Limited published case reports discuss pregnancies in women taking cholic acid for 3β-HSD deficiency resulting in healthy infants. These reports may not adequately inform the presence or absence of drug-associated risk with the use of CHOLBAM during pregnancy. The background risk of major birth defects and miscarriage for the indicated population is unknown. However, the background risk in the U.S. general population of major birth defects is 2-4% and of miscarriage is 15-20% of clinically recognized pregnancies. 8.2 Lactation Risk Summary Endogenous cholic acid is present in human milk. Clinical lactation studies have not been conducted to assess the presence of CHOLBAM in human milk, the effects of CHOLBAM on the breastfed infant, or the effects of CHOLBAM on milk production. There are no animal lactation data and no data from case reports available in the published literature.

Pharmacokinetics

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Metabolism
Orally administered cholic acid is subject to the same metabolic pathway as endogenous cholic acid. Cholic acid is absorbed by passive diffusion along the length of the gastrointestinal tract.

Overdosage

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Concurrent elevations of serum GGT and ALT may indicate CHOLBAM overdose. If an overdose is suspected, discontinue CHOLBAM and treat symptoms. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline [see Dosage and Administration ( 2.2 ) and Warnings and Precautions ( 5.1 )] .

Approval history

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  • Mar 17, 2015NDANDA205750Mirum

FAERS reports

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Reference statistics only. FAERS reports are voluntarily submitted and are not incidence rates, safety signals, or causal evidence. Counts reflect reporting volume — how often a reaction was reported, not how often it occurs. For decision-grade use, consult openFDA and the FAERS Public Dashboard directly.
397 total reports matchedLatest report Share = reports listing the reaction ÷ total matched reports. Rows can sum to >100% because a single report often lists multiple reactions.
  1. 1Diarrhoea5614%
  2. 2Death4010%
  3. 3Vomiting297.3%
  4. 4Pyrexia215.3%
  5. 5Seizure205.0%
  6. 6Constipation194.8%
  7. 7Aspartate Aminotransferase Increased164.0%
  8. 8Disease Progression164.0%
  9. 9Alanine Aminotransferase Increased153.8%
  10. 10Abdominal Pain133.3%
  11. 11Cough133.3%
  12. 12Nasopharyngitis133.3%
  13. 13Decreased Appetite102.5%
  14. 14Gastrooesophageal Reflux Disease102.5%
  15. 15Ear Infection92.3%

Literature

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Recent PubMed references pinned to Cholic Acid as a MeSH major topic. Citations link to pubmed.ncbi.nlm.nih.gov.

Clinical trials

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The 10 most recently updated of 199 ClinicalTrials.gov registrations naming Cholic Acid as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

Frequently asked questions

How does Cholic Acid work?
Cholic acid is a primary bile acid synthesized from cholesterol in the 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.
What is Cholic Acid used for?
According to FDA labeling, Cholic Acid carries indications including: CHOLBAM is a bile acid indicated for: • Treatment of bile acid synthesis disorders due to single enzyme defects (SEDs). ( 1.1 ) • Adjunctive treatment of peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.
What class of drug is Cholic Acid?
Cholic Acid is classified as Bile acids and derivatives, Bile Acid.
What are the brand names for Cholic Acid?
Cholic Acid is marketed under brand names including Cholbam.
What are the contraindications for Cholic Acid?
Cholic Acid labeling lists contraindications including: None.. Always consult the full prescribing information and a clinician.
Note. Data for cholic-acid is illustrative MVP content compiled from public sources. pharmacopeia is for educational and informational use only and is not a substitute for professional medical advice.

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