The FDA has approved a new drug called Cholbam (cholic acid) for two rare genetic conditions: bile acid synthesis disorders and certain peroxisomal disorders like Zellweger spectrum disorders. These conditions can cause serious liver problems and trouble absorbing fat and vitamins. Cholbam is a bile acid that replaces the ones the body cannot make, helping the liver work better and improving fat absorption. This is the first FDA approved treatment for these often fatal diseases.
Cholbam is for patients who have liver disease, fatty stools, or problems from not absorbing fat soluble vitamins. It is taken by mouth and must be used under a doctor's care. Doctors will check liver function regularly and stop the drug if there is no improvement after three months.
The approval gives patients a targeted therapy for a previously untreatable condition. However, the drug has not been shown to help with symptoms outside the liver. Patients should talk to their doctor about whether Cholbam is right for them and what to expect from treatment.