The Food and Drug Administration approved cholic acid capsules for treatment of children aged 3 weeks and older and adults with bile acid synthesis disorders due to single enzyme defects. It is the first drug approved for this indication, the agency announced.
The bile acid also was approved as 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. Cholic acid will be marketed as Cholbam by Asklepion Pharmaceuticals, the FDA said in its statement.
Approval was based on uncontrolled studies, which evaluated treatment of patients, mostly children, over a period of 18 years. In the study of 62 patients with bile acid synthesis disorders due to single enzyme defects, 64% of patients with evaluable data responded with improvements in baseline liver function tests and weight; two-thirds of patients survived longer than 3 years. In the study of cholic acid for the treatment of peroxisomal disorder in 31 children, improvements in baseline liver function tests and weight were noted in 46%; 42% of patients survived longer than 3 years.
Diarrhea was the most common adverse event associated with treatment. “The use of Cholbam should be carefully monitored by an experienced hepatologist or pediatric gastroenterologist, and treatment discontinued in patients developing worsening liver function,” according to the FDA statement.
The manufacturer is required to conduct a postapproval observational study to evaluate the long-term safety of cholic acid.
The prescribing information is available at www.accessdata.fda.gov/drugsatfda_docs/label/2015/205750s000lbl.pdf.