FDA approves first-ever med for rare bile disorder
- Cholbam (cholic acid) capsules, developed by Asklepion Pharmaceuticals, are the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single-enzyme defects, and for patients with peroxisomal disorders.
- These types of disorders occur when the enzymes needed to synthesize cholic acid are missing. Cholic acid is normally produced in the liver from cholesterol. The result of untreated bile acid synthesis disorders is failure to grow and a potentially life-threatening liver injury.
- The indication for Cholbam, which is an oral treatment, is for children aged three weeks and older and for adults.
Although the prevalence of bile acid synthesis and peroxisomal disorders is rare, this group of diseases can be deadly and affects people across the age spectrum. The FDA gave Asklepion a rare pediatric disease priority review voucher in order to support and expedite development.
Cholbam's efficacy for treating patients with bile acid synthesis disorders due to single-enzyme defects was assessed in an 18-year uncontrolled trial involving 50 patients, with an extension period which allowed researchers to cull an additional 21 months of data on 12 of the subjects. Overall, treatment improved baseline liver function in 64% of evaluated patients, who ranged in age from 3 weeks to 36 years at the beginning of the trial.
Separately, researchers evaluated Cholbam in 29 patients with peroxisomal patients over an 18-year period. Overall, 46% of patients responded and 42% of patients survived for three years or more. The most common side effect in both studies was diarrhea.
The FDA is requiring post-marketing studies to assess the long-term safety of Cholbam.