Cholic acid

(Cholbam®)

Cholbam®

Drug updated on 5/17/2024

Dosage FormCapsule (oral; 50 mg, 250 mg)
Drug ClassEnzymes
Ongoing and
Completed Studies
ClinicalTrials.gov

Indication

  • Indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs).
  • Indicated for 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.

Summary
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  • Cholic acid (Cholbam) is indicated for the treatment of bile acid synthesis disorders due to single enzyme defects and as an 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.
  • Two randomized controlled studies provided information about the safety and effectiveness of Cholic acid (Cholbam).
  • The studies included 53 patients with either single enzyme defects or Zellweger spectrum disorders. Notable improvements were observed in urinary bile acids, body height, and body weight for these patients, especially those who had not been previously treated with cholic acid.
  • In terms of safety profile, upper respiratory tract infection was reported as a common adverse event among 17% of the participants, indicating that Cholbam has a tolerable safety profile.
  • For the Zellweger Spectrum Disorders patient population comprising 17 individuals continuing oral cholic acid treatment for an additional 12 months, totaling 21 months; while it effectively suppressed bile acids synthesis reducing levels of toxic C27-bile acids intermediates in plasma, no significant improvement was noted after this period, raising concerns about potential hepatotoxicity despite the absence of specific treatment-emergent adverse events mentioned in this subgroup.
  • Compared to other treatments for BASD and PDs, specifically ZSDs, Cholbam demonstrates robust efficacy ameliorating symptoms and biochemical markers associated with these disorders, particularly improving liver function and promoting growth in BASD. However, its application requires careful consideration given the non-improvement in clinical parameters and potential hepatotoxicity risks in cases of advanced liver disease.

Product Monograph / Prescribing Information

Document TitleYearSource
Cholbam (cholic acid) Prescribing Information.2020Travere Therapeutics., San Diego, CA

Randomized Controlled Trials

Document TitleSex DistributionYearSource
Open-label phase 3 continuation study of cholic acid in patients with inborn errors of bile acid synthesis.
53Subjects
F: 43%
M: 57%
2020Journal of Pediatric Gastroenterology and Nutrition
The cholic acid extension study in Zellweger spectrum disorders: Results and implications for therapy
Data not availableSubjects
F: null%
M: null%
2019Journal of Inherited AMetabolic Disease

Sex Distribution:

F:43%
M:57%
53Subjects

Year:

2020

Source:Journal of Pediatric Gastroenterology and Nutrition


Sex Distribution:

No Data

Year:

2019

Source:Journal of Inherited AMetabolic Disease