FDA Approves Treatment for Bile Acid Synthesis Disorders

Patients with bile acid synthesis and peroxisomal disorders will soon have access to the first FDA-approved treatment option.

The FDA today approved cholic acid (Cholbam) capsules for pediatric and adult patients who have bile acid disorders due to single enzyme defects. It was also approved for treating patients with peroxisomal disorders, such as Zellweger spectrum disorders.

These genetic, metabolic conditions can bring about liver disease, steatorrhea, and complications from decreased fat-soluble vitamin absorption, according to the FDA.

Cholbam was examined in a study of 50 patients with bile acid synthesis disorders, and 64% responded to treatment with improvements in baseline liver function tests and weight. Two-thirds of the patients survived >3 years, as well. Cholbam’s efficacy was also tested in a trial involving 29 patients who had peroxisomal disorders. About 45% of patients responded to treatment with improvements in their baseline liver function tests and weight, and 42% of patients lived for >3 years.

Cholbam can be taken orally among patients ≥3 weeks old. The most common side effect was diarrhea.

“This approval underscores the agency’s commitment to making treatments available to patients with rare diseases,” Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research, said in a press release. “Prior to today’s approval, patients with these rare bile acid synthesis disorders had no approved treatment options.”