Cholic acid, a drug used to treat bile acid synthesis disorders and peroxisomal disorders, has been approved by the US Food and Drug Administration (FDA).
Marketed as Cholbam by Asklepion Pharmaceuticals, cholic acid is the first drug to be available in the United States to treat patients with these rare, genetic, metabolic conditions who lack the enzymes needed to synthesise cholic acid, normally produced in the liver from cholesterol.
The capsules will be available for adults and children over the age of three weeks following an FDA priority review, conducted because of the drug’s potential to treat rare childhood diseases.
Approval of cholic acid for treatment of bile acid synthesis disorders caused by single enzyme defects follows a single-arm trial involving 50 patients who were treated for 18 years. Patients had an average age of four years at the start of treatment. A further trial followed 21 of these patients and an additional 12 patients for another 21 months. In 64% of patients for whom data were available, two-thirds survived longer than three years.
FDA approval for the treatment of peroxisomal disorders follows a single-arm trial involving 29 patients — with an average age of under two years at the start — who were treated for 18 years. An extension trial followed ten of these patients, as well as another two patients for another 21 months. Response to treatment was noted in 46% of patients with evaluable data; 42% of whom survived more than three years.
The drug is already approved for use in Europe.