First Drug Approved for Rare Bile Acid Synthesis Disorders

The Food and Drug Administration (FDA) has approved Cholbam (cholic acid; Asklepion) capsules for the treatment of patients ≥3 weeks old with bile acid synthesis disorders due to single enzyme defects, and as adjunct to standard of care for patients with peroxisomal disorders (including Zellweger spectrum disorders).

Cholbam is the first FDA-approved medication to turn off a genetically-damaged bile synthesis pathway and prevent its toxic products from damaging the liver. It works by down regulating cholesterol 7alpha-hydroxylase, the rate limiting step in bile acid synthesis. This inhibits the production and accumulation of hepatotoxic and cholestatic bile acid precursors, stimulates bile flow and secretion, and facilitates fat absorption.

The approval of Cholbam for the treatment of patients with bile acid synthesis disorders due to single enzyme defects was supported by data from an uncontrolled trial (n=50) that treated patients over an 18 year period. An extension trial followed 21 of these patients and another 12 were enrolled. Treatment response was evaluated by improvements in baseline liver function test and weight. Responses were seen in 64% of patients with evaluable data and two-thirds of patients had >3 year survival.

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The approval of Cholbam for the treatment of patients with peroxisomal disorders, including Zellweger spectrum disorders, was supported by data from an uncontrolled trial (n=29) that treated patients over an 18 year period. An extension trial followed 10 of these patients and another two were enrolled. Treatment response was evaluated by improvements in baseline liver function tests and weight. Responses were seen in 46% of patients with evaluable data and 42% of patients had >3 year survival.

Cholbam was granted a rare pediatric disease priority review voucher. The FDA is requiring an observational study to assess the long-term safety of Cholbam.

For more information visit FDA.gov.

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