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.