Drug updated on 3/28/2024
| Dosage Form | Capsule (oral; 50 mg, 250 mg) |
| Drug Class | Bile acids |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- For treatment of bile acid synthesis disorders due to single enzyme defects (SEDs).
- 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
- Cholic acid (Cholbam) is used for the treatment of bile acid synthesis disorders due to single enzyme defects and as an adjunctive treatment for peroxisomal disorders including Zellweger spectrum disorders in patients who exhibit manifestations of liver disease, steatorrhea or complications from decreased fat-soluble vitamin absorption.
- A total of one document was reviewed which was a Randomized Controlled Trial that studied the efficacy and safety profile of cholic acid (Cholbam).
- The study included 53 patients with bile acid synthesis disorder; 41 had single enzyme defects while 12 had Zellweger spectrum disorders.
- Of these participants, 22 were newly diagnosed and started on cholic acid therapy during this trial whereas remaining 31 were already receiving cholic acid from a previous study.
- Significant improvements in urinary bile acids, height, body weight were observed post-treatment with Cholic Acid (P=0.003), indicating its effectiveness in treating BASD conditions.
- The most common adverse event reported during the trial period was upper respiratory tract infection affecting about 17% participants but overall it suggests that oral administration of Cholic Acid provides safe short- and long-term therapy for patients suffering from Bile Acid Synthesis Disorders.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Cholbam (cholic acid) Prescribing Information. | 2020 | Travere Therapeutics, San Diego, CA |
Randomized Controlled Trials
| Document Title | Sex Distribution | Year | Source |
|---|---|---|---|
| Open-label phase 3 continuation study of cholic acid in patients with inborn errors of bile acid synthesis. | 53Subjects F: 43% M: 57% | 2020 | Journal of Pediatric Gastroenterology and Nutrition |
Document Title
Sex Distribution:
F:43%
M:57%
53Subjects
Year:
2020
Source:Journal of Pediatric Gastroenterology and Nutrition