Odevixibat for cholestatic liver disease in Alagille syndrome patients

Odevixibat is currently in clinical development for the treatment of cholestatic liver disease in Alagille syndrome (ALGS) patients. ALGS is an inherited developmental disorder, caused by a mutation in the JAGGED1 gene or in the NOTCH2 gene. The mutation causes problems with early embryonic development leading to abnormalities in various parts of the body. ALGS is a rare multisystem disorder with a wide variety of clinical manifestations affecting the liver, heart, skeleton, eyes, central nervous system, kidneys, and facial features. Most patients with this disorder have liver abnormalities resulting from having too few bile ducts as well as other physiological indicators such as a small chin or deep-set eyes. Due to the reduced number of ducts, bile acids build up in the liver and damage the liver tissue as well as causing severe itching which can significantly limit quality of life and sleep. ALGS is a long-term debilitating and life-threatening disease that has no approved treatments, and many patients require liver transplants before adulthood.