Summary

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• Cholbam (cholic acid) is indicated for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and 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.
• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• Statistically significant improvements in urinary atypical bile acids (P = 0.003) and in height and body weight (P < 0.001) were observed in patients with bile acid synthesis disorders (BASD), particularly in treatment-naïve individuals, based on baseline-to-best postbaseline analyses.
• In patients with Zellweger spectrum disorders (ZSD), suppression of bile acid synthesis and reduction of toxic C27-bile acid intermediates were maintained for 21 months, but no improvement was observed in clinically relevant outcome measures after 9 and 21 months.
• Serum liver chemistries showed a trend towards improvement in alanine aminotransferase and aspartate aminotransferase levels in treatment-naïve patients with BASD, with stable levels in previously treated patients, while no significant changes in liver tests or liver elasticity were observed in ZSD patients.
• The most common treatment-emergent adverse event (TEAE) was upper respiratory tract infection, occurring in 17% of patients.
• Caution was advised in patients with advanced liver disease due to potential hepatotoxicity, although no significant changes were noted in liver tests or liver elasticity.
• The reviewed studies include patients with bile acid synthesis disorders (n = 41) and Zellweger spectrum disorders (n = 29), highlighting differences in outcomes between treatment-naïve and previously treated patients; no population types or subgroups information beyond this is available.