Asklepion Pharmaceuticals, LLC. today announced that the U.S. Food and Drug Administration (FDA) approved CHOLBAM™ (cholic acid) as a once-daily treatment for cholic acid deficiency in bile acid synthesis disorders due to single enzyme defects. This indication is based on CHOLBAM's ability to markedly improve or normalize liver function test values and to improve general health, as measured by weight gain. CHOLBAM was also approved as an adjunct to standard of care for peroxisomal disorders including Zellweger spectrum disorders in patients with evidence of liver disease, based on improvements in liver function.
CHOLBAM is the first medication approved by the FDA to turn off a genetically-damaged bile synthesis pathway and prevent its toxic products from damaging the liver. The approval of CHOLBAM is a testament to the pioneering work of Dr. Kenneth Setchell and Dr. James Heubi of Cincinnati Children's Hospital and Medical Center, who were the first to recognize many of the constituent diseases and develop a safe and effective therapy. The U.S. approval of CHOLBAM was based on two pivotal trials that showed improved liver function test values and restoration of growth assessed by weight gain in comparison to the natural history of untreated patients. Some patients in these trials have been healthy on therapy, exhibiting normal liver function for more than 16 years.