This notice was prepared by Dale Whitby, PharmD and Sharon Williams, PharmD (PPAG Members)
On March 17, 2015 the FDA approved cholic acid (Cholbam) for the treatment of patients with bile acid synthesis disorders due to single enzyme defects and for patients with peroxisomal disorders (including Zellweger spectrum disorders). Cholbam is a once- or twice-daily oral therapy approved for use in patients 3 weeks of age and older. The manufacturer of Cholbam, Asklepion Pharmaceuticals LLC, was granted a rare pediatric disease priority review voucher; Cholbam is the first FDA-approved therapy for the treatment of bile acid synthesis disorders due to single enzyme defects.
The most common adverse events associated with cholic acid use are GI effects, with diarrhea being the most frequent. Laboratory monitoring during treatment initiation and maintenance is required, and cholic acid therapy should be discontinued if hepatic function worsens.
The FDA approval of Cholbam was based on the results of a long-term trial followed by an extension trial that showed improvements in baseline liver function tests (LFTs) and growth. An uncontrolled trial involving 50 patients with bile acid synthesis disorders treated over an 18-year period (plus a 21-month extension that included 21 of the original patients plus 12 new patients) showed a positive response in 64% of patients with evaluable data. Twenty-nine patients with Zellweger spectrum disorders were also included (the extension trial included 10 of the original patients plus two new patients). A response was noted in 46% of patients.
Cholbam will be marketed as 50-mg and 250-mg capsules that can be opened and mixed with milk or soft food to facilitate administration in infants and young children.
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