FDA Approves Cholbam to Treat Rare Bile Acid Synthesis Disorders

The Food and Drug Administration March 17 approved Cholbam (cholic acid) capsules, the first treatment approved by the agency for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for patients with peroxisomal disorders (including Zellweger spectrum disorders).

Patients with these rare, genetic, metabolic conditions exhibit manifestations of liver disease, steatorrhea (presence of fat in the stool) and complications from decreased fat-soluble vitamin absorption, the FDA said. Individuals with these disorders lack the enzymes needed to synthesize cholic acid, a primary bile acid normally produced in the liver from cholesterol. The absence of cholic ...