FDA Approves Cholbam (cholic acid) for Bile Acid Synthesis Disorders and Peroxisomal Disorders
The FDA has approved Cholbam (cholic acid) for the treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and as adjunctive treatment for peroxisomal disorders (PDs) including Zellweger spectrum disorders in patients with liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. This is the first FDA-approved treatment for these rare, often fatal conditions. Cholbam is a bile acid that replaces deficient bile acids, improving liver function and fat absorption. The approval provides a targeted therapy for patients with these inherited metabolic disorders, though the label notes that safety and effectiveness on extrahepatic manifestations have not been established. Clinicians should monitor liver function closely and discontinue if no improvement within 3 months.
＋ Clinical Details (Mechanism · Dosing · Trial Data · Warnings)
Cholbam (cholic acid) is a bile acid that replaces deficient bile acids in patients with bile acid synthesis disorders due to single enzyme defects and peroxisomal disorders. It promotes bile flow and absorption of fat-soluble vitamins.
Cholbam is indicated for treatment of bile acid synthesis disorders due to single enzyme defects (SEDs) and as 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. Limitations: Safety and effectiveness on extrahepatic manifestations have not been established.
Recommended dosage: 10 to 15 mg/kg once daily or in two divided doses, in pediatric patients and adults. For patients with concomitant familial hypertriglyceridemia: 11 to 17 mg/kg once daily or in two divided doses, adjusted based on clinical response. Administer with food. Capsules can be opened and mixed with drink/food for patients unable to swallow. Monitor AST, ALT, GGT, alkaline phosphatase, bilirubin, and INR monthly for first 3 months, every 3 months for next 9 months, every 6 months for next 3 years, then annually. Discontinue if liver function does not improve within 3 months, if complete biliary obstruction develops, or if persistent worsening of liver function or cholestasis occurs.
Trial data not available in label.
Discontinue if liver function does not improve within 3 months, if complete biliary obstruction develops, or if persistent clinical or laboratory indicators of worsening liver function or cholestasis occur. Concurrent elevations of serum GGT and serum ALT may indicate overdose. Monitor liver function and consider restarting at a lower dose when parameters return to baseline.
Cholbam is a first-line replacement therapy for bile acid synthesis disorders due to SEDs and adjunctive treatment for peroxisomal disorders. It should be initiated and monitored by an experienced hepatologist or pediatric gastroenterologist. It addresses liver disease and fat malabsorption but has not been shown to affect extrahepatic manifestations.
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