The FDA approved Cholbam, an oral capsule of cholic acid marketed by Baltimore, Maryland-based Asklepion Pharmaceuticals LLC for two rare metabolic diseases (bile acid synthesis disorders and peroxisomal disorders). These two rare metabolic diseases are caused by genetic mutations in a group of genes encoding enzymes involved in the biosynthesis of cholic acid, a major component of bile acid and proteins responsible for peroxisome biogenesis.  The approval is based on a single arm trial involving 50 patients treated over an 18 year period and literature.  In this trial, 64% of patients showed improvements in baseline liver function tests and weight.  This is the first medication approved by the FDA for this group of orphan diseases.


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