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Innovation Observatory > Reports > Drugs > Maralixibat for cholestatic liver disease in patients with Alagille syndrome

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Maralixibat for cholestatic liver disease in patients with Alagille syndrome


Gastrointestinal, Pancreatic and Liver Disease

February 2021

Maralixibat is in clinical development for the treatment of Alagille syndrome. Alagille syndrome is an inherited developmental disorder, caused by a mutation in the JAG1 gene or in the Notch2 gene. The mutation causes problems with early embryonic development leading to abnormalities in various parts of the body. Most patients with this disorder have liver abnormalities resulting from having too few bile ducts. 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. Alagille syndrome is a long-term debilitating and life-threatening disease that has no approved treatments and many patients require liver transplants before adulthood.
Maralixibat is an orally administered drug that blocks certain channels called ileal bile acid transporters through which the bile acids leave the intestine to reach the blood vessels that carry them back to the liver. By blocking these channels, maralixibat is expected to help reduce the amount of toxic bile acids in the liver, thereby reducing the itching and other quality of live measures in patients with Alagille syndrome. If licensed, maralixibat will provide a treatment option for patients with Alagille syndrome, a disease of unmet clinical need.

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