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 (PBD-ZSD), 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 PBD-ZSD, 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

Why should I prescribe CHOLBAM®?

Treatment with CHOLBAM® may help reduce liver injury. CHOLBAM® may improve bile flow and increase solubilization of dietary fats and fat-soluble vitamins through emulsification.3,4

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 tests are available for BASD patients?

There are 2 tests available at no cost to qualifying patients:

  1. PreventionGenetics 77-gene cholestasis panel
    In partnership with PreventionGenetics, Travere Therapeutics offers a no-cost testing program for qualifying patients to identify the genetic causes of cholestasis.
  2. Mass spectrometry at Cincinnati Children’s Hospital Medical Center
    This urinary bile acid profile test by fast atom bombardment mass spectrometry (FAB-MS) is performed exclusively at Cincinnati Children’s Hospital Medical Center and confirms defects in bile acid synthesis in BASD through the presence of atypical bile acids.

Learn more about these tests.

Bile acid synthesis disorder (BASD)

What is BASD?

BASD due to single enzyme defects involves congenital deficiencies in enzymes required for catalyzing key reactions in the synthesis of the 2 main bile acids—cholic acid (CA) and chenodeoxycholic acid (CDCA).5

Often present in the first few months of life, BASD can produce life-threatening, progressive cholestatic liver disease.6,7

How does BASD present clinically?

BASD, due to single enzyme defects, has distinct clinical features. Symptoms of BASD may include4,8,9:

  • Cholestatic jaundice
  • Acholic stools
  • Hepatomegaly
  • Failure to thrive
  • Absence of pruritus

Read more about BASD.

How do I establish a clinical diagnosis of BASD?

Lab abnormalities that may be present include3,4,8:

  • Elevated bilirubin (mg/dL)
  • Elevated ALT/AST (U/L)
  • GGT (U/L) ≤150
  • Normal to low primary bile acids (serum) (µmol/mL)

If lab abnormalities are present, diagnose with genetic testing or urine bile acid profile.

Learn more about these tests.

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 in the HUB?

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. Braverman NE, Raymond GV, Rizzo WB, et al. Mol Genet Metab. 2016:117(3):313-321. doi: 10.1016/j.ymgme.2015.12.009.  3. Setchell KDR. Defects in bile acid synthesis – specific and treatable causes of metabolic liver disease. Adolf Windaus Prize Lecture. 2004.  4. Rinawi F, Iancu TC, Hartman C, et al. Fat malabsorption due to bile acid synthesis defect. Isr Med Assoc J. 2015;17(3):190-192.  5. Heubi JE, Setchell KDR, Bove KE. Inborn errors of bile acid metabolism. Clin Liver Dis. 2018;22(4):671-687. doi: 10.1016/j.cld.2018.06.006.  6. Bove KE, Heubi JE, Balistreri WF, Setchell KD. Bile acid synthetic defects and liver disease: a comprehensive review. Pediatr Dev Pathol. 2004;7:315-334. doi: 10.1007/s10024-002-1201-8.  7. Clayton PT. Disorders of bile acid synthesis. J Inherit Metab Dis. 2011;34:593-604. doi: 10.1007/s10545-010-9259-3.  8. Sundaram SS, Bove KE, Lovell MA, Sokol RJ. Mechanisms of disease: inborn errors of bile acid synthesis. Nat Clin Pract Gastroenterol Hepatol. 2008;5(8):456-468. doi: 10.1038/ncpgasthep1179.  9. Gonzales E, Gerhardt MF, Fabre M, et al. Oral cholic acid for hereditary defects of primary bile acid synthesis: a safe and effective long-term therapy. Gastroenterology. 2009;137(4):1310-1320. doi: 10.1053/j.gastro.2009.07.043.

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.