Frequently asked questions

About CHOLBAM®

When may CHOLBAM® (cholic acid) capsules be appropriate?

CHOLBAM® (cholic acid) is a bile acid indicated for:

  • Treatment of bile acid synthesis disorders due to single enzyme defects1
  • Adjunctive treatment of peroxisomal disorders, including peroxisomal biogenesis disorder-Zellweger spectrum disorder (PBDZSD), in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption.1

The safety and effectiveness of CHOLBAM® on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including PBDZSD, have not been established.1

How does CHOLBAM® work?

CHOLBAM® (cholic acid) capsules work by restoring cholic acid levels. By replacing cholic acid in the hepatocyte, CHOLBAM® is thought to suppress the production of the hepatotoxic atypical bile acids DHCA and THCA.2

CHOLBAM® may improve bile flow and increase solubilization of dietary fats and fat-soluble vitamins through emulsification.2

Why should I prescribe CHOLBAM®?

Treatment with CHOLBAM® may reduce hepatocyte exposure to DHCA and THCA and reduce liver injury. CHOLBAM® may also increase intraluminal bile acid concentrations to improve absorption of fats and fat-soluble vitamins to facilitate weight gain.2

How do I dose CHOLBAM®?

Starting dose of CHOLBAM® for both children and adults should be based on weight. The recommended dose of CHOLBAM® is 10 to 15 mg/kg once a day, which can be divided into 2 doses if necessary.1

How is CHOLBAM® administered?

CHOLBAM® should be taken with food. For infants and children who cannot swallow capsules, CHOLBAM® capsules can be opened and mixed with either infant formula or breast milk (for younger children), or soft food such as mashed potatoes or apple puree (for older children and adults) in order to mask any unpleasant taste.1

How should I monitor patients taking CHOLBAM®?

Monitor AST, ALT, serum GGT, ALP, bilirubin, and INR every month for the first 3 months, every 3 months for the next 9 months, every 6 months during the subsequent 3 years, and annually thereafter. Learn more about monitoring.1

What are potential adverse events with CHOLBAM®?

In the CHOLBAM® clinical trials, diarrhea was the most common adverse reaction in approximately 2% of the patient population. All other adverse reactions occurred in ≤1% of the patient population.1 See more safety information.

Testing

What test can confirm diagnosis of PBDZSD?

Early diagnosis and treatment are important to help reduce the risk of liver damage.

In partnership with PreventionGenetics, Travere Therapeutics offers a no-cost genetic testing program for qualifying patients to help identify PBDZSD, a rare autosomal recessive disorder caused by mutations in one of 13 PEX genes and characterized by an impaired peroxisome assembly, leading to multiple enzyme deficiencies. Learn more about this test.

What test can determine the extent of liver involvement in PBDZSD patients?

Travere Therapeutics has partnered with Cincinnati Children’s Hospital Medical Center to offer liquid chromatography–mass spectrometry analysis of bile acid profile at no cost to qualifying patients.

This test will help identify the presence and concentration levels of DHCA/THCA, atypical bile acids that can be hepatotoxic and associated with liver disease. Learn more about this test.

Peroxisomal biogenesis disorder-Zellweger spectrum disorder (PBDZSD)

What is peroxisomal biogenesis disorder-Zellweger spectrum disorder (PBDZSD)?

PBDZSD is a heterogeneous group of autosomal recessive disorders characterized by a defect in peroxisome formation, affecting multiple metabolic and biosynthetic processes. PBDZSD is caused by pathogenic variants in one of 13 PEX genes.3

Previously regarded as 3 distinct disorders, PBDZSD is now considered to comprise 1 disease that ranges in phenotype from severe (previously referred to as Zellweger syndrome) to moderate (previously referred to as neonatal adrenoleukodystrophy or NALD) to mild (previously referred to as infantile Refsum disease or IRD).4,5

How does PBDZSD present?

In affected patients, the severity and range of signs and symptoms vary according to subgroup, based on age at presentation. Signs and symptoms also can vary by patient within severity subgroups, ie, not every patient will present the same way, even if they are grouped as all severe, all moderate, or all mild.3,4

Patients with milder signs and symptoms may have a delay in diagnosis, as these signs and symptoms may not appear abnormal to parents. In addition, many other diseases present similarly. Overall, common presenting signs include developmental delay and other neurologic abnormalities, vision impairment (due to retinal degeneration), sensorineural hearing loss, and evidence of liver disease.3,5-7

Patient Resources

What is the CHOLBAM® Total Care HUB?

The Total Care HUB is a comprehensive support program for you, your patients taking CHOLBAM®, and their caregivers. Patients will connect with a dedicated team of professionals to assist them throughout their treatment. Learn more about this valuable program.

How do I enroll my patients?

When you prescribe CHOLBAM® to your patients, they will be automatically enrolled in the Total Care HUB. Click here to access the form.

REFERENCES:  1. CHOLBAM® (cholic acid) capsules, for oral use [prescribing information]. San Diego, CA: Travere Therapeutics, Inc.; April 2021.  2. Berendse K, Klouwer FCC, Koot BGP, et al. Cholic acid therapy in Zellweger spectrum disorders. J Inherit Metab Dis. 2016;39(6):859-868. doi: 10.1007/s10545-016-9962-9.  3. Braverman NE, Raymond GV, Rizzo WB, et al. Peroxisome biogenesis disorders in the Zellweger spectrum: An overview of current diagnosis, clinical manifestations, and treatment guidelines. Mol Genet Metab. 2016:117(3):313-321. doi: 10.1016/j.ymgme.2015.12.009.  4. Klouwer FCC, Berendse K, Ferdinandusse S, Wanders RJA, Engelen M, Poll-The BT. Zellweger spectrum disorders: clinical overview and management approach. Orphanet J Rare Dis. 2015;10:151. doi: 10.1186/s13023-015-0368-9.  5. Braverman NE, D’Agostino MD, Maclean GE. Peroxisome biogenesis disorders: biological, clinical and pathophysiological perspectives. Dev Disabil Res Rev. 2013;17(3):187-196. doi: 10.1002/ddrr.1113.  6. Zellweger H, Maertens P, Superneau D, Wertelecki W. History of the cerebrohepatorenal syndrome of Zellweger and other peroxisomal disorders. South Med J. 1988;81(3):357-364. doi: 10.1097/00007611-198803000-00017.  7. Poll-The BT, Saudubray JM, Ogier HA, et al. Infantile Refsum disease: an inherited peroxisomal disorder. Comparison with Zellweger syndrome and neonatal adrenoleukodystrophy. Eur J Pediatr. 1987;146:477-483. doi: 10.1007/BF00441598.

INDICATION

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

LIMITATIONS OF USE

The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment

  • Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment.
  • Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose.
  • Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis.

ADVERSE REACTIONS

  • The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy.

DRUG INTERACTIONS

  • Inhibitors of Bile Acid Transporters: Avoid concomitant use of inhibitors of the bile salt efflux pump (BSEP) such as cyclosporine. Concomitant medications that inhibit canalicular membrane bile acid transporters such as the BSEP may exacerbate accumulation of conjugated bile salts in the liver and result in clinical symptoms. If concomitant use is deemed necessary, monitoring of serum transaminases and bilirubin is recommended.
  • Bile Acid Binding Resins: Bile acid binding resins such as cholestyramine, colestipol, or colesevelam adsorb and reduce bile acid absorption and may reduce the efficacy of CHOLBAM. Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after a bile acid binding resin.
  • Aluminum-Based Antacids: Aluminum-based antacids have been shown to adsorb bile acids in vitro and can reduce the bioavailability of CHOLBAM. Take CHOLBAM at least 1 hour before or 4 to 6 hours (or at as great an interval as possible) after an aluminum-based antacid.

PREGNANCY

No studies in pregnant women or animal reproduction studies have been conducted with CHOLBAM. Women who become pregnant during CHOLBAM treatment are encouraged to call 1-844-202-6262.

LACTATION

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. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CHOLBAM and any potential adverse effects on the breastfed infant from CHOLBAM or from the underlying maternal condition.

GERIATRIC USE

It is not known if elderly patients respond differently from younger patients.

HEPATIC IMPAIRMENT

  • Discontinue treatment with CHOLBAM if liver function does not improve within 3 months of the start of treatment.
  • Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline.

OVERDOSAGE

Concurrent elevations of serum GGT and serum ALT may indicate CHOLBAM overdose. In the event of overdose, the patient should be monitored and treated symptomatically. Continue to monitor laboratory parameters of liver function and consider restarting at a lower dose when the parameters return to baseline.
To report SUSPECTED ADVERSE REACTIONS, contact Travere Therapeutics at 1‑877‑659‑5518 or FDA at 1‑800‑FDA‑1088 or www.fda.gov/medwatch.
Please see accompanying full Prescribing Information for additional Important Safety Information.