FDA approves cholic acid for bile acid synthesis disorders

FDA has approved cholic acid (Cholbam), a once-daily treatment for pediatric and adult patients who have bile acid synthesis disorders due to single enzyme defects. It is also approved as an adjunct to standard care for patients with peroxisomal disorders, such as Zellweger spectrum disorders.

This is the first medication that the FDA has approved to help combat a genetically-damaged bile synthesis pathway, according to its manufacturer, Asklepion Pharmaceuticals.

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In two clinical trials, cholic acid was shown to improve liver function test values and restore growth in patients based on weight gain compared to individuals not treated with the new therapy. Some trial patients were able to achieve normal liver function and stay healthy on treatment for more than 16 years, noted the manufacturer.

“The FDA approval of Cholbam is of vital importance to patients and families who are dealing with bile acid deficiency diseases,” said Philip Rosenthal, MD, director of pediatric hepatology at the University of California, San Francisco. “Making this therapy more widely available is a significant accomplishment of science and collaboration in this rare disease space.”

Approximately 500 children are born in the United States each year and diagnosed with bile acid deficiencies. If they are not treated, these children develop cholestasis, nutrient malabsorption, liver disease, neurological problems, and possibly liver failure, leading to death. Zellweger spectrum disorder occurs in one of 50,000 newborns, and approximately 80% will develop liver disease.

Cholic acid can prevent damage to the liver by preventing the production of toxic products in patients with bile acid deficiency diseases. “Cholic acid is one of the primary bile acids on which essential physiological functions depend,” according to the manufacturer.

The drug is approved as an oral treatment for children aged three weeks and older and for adults. The recommended dosage for children who are 1 month and older and for adults is 10 to 15 mg/kg once daily or administered in two divided doses.

The manufacturer warns that patients should be closely monitored when starting therapy. Liver function tests, fat soluble vitamin levels, and an INR test or prothrombin time are recommended. If the patient develops concurrent elevations of serum gamma glutamyl transferase and serum alanine aminotransferase, this indicates an overdose.

After initial drug adjustment, patients should be monitored annually for serum and urine bile acids and liver function, and the dose of cholic acid should be adjusted, if necessary.

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