Clinical development of the drug for the treatment of hypercholesterolaemia, primary biliary cholangitis (PBC) and primary sclerosing cholangitis has been discontinued. Maralixibat has Orphan Drug designation for the treatment of ALGS, PFIC and biliary atresia from both the US FDA and the European Medicines Agency. Maralixibat is also under regulatory review for ALGS in Europe, and clinical development for cholestatic liver disorders including ALGS, PFIC and biliary atresia is continuing in several other countries. Treatment should be initiated at a dosage of 190 µg/kg once daily with an increase to 380 µg/kg once daily after 1 week, as tolerated (maximum daily dose of 28.5 mg). Maralixibat is available as an oral solution and should be taken 30 min before the first meal of the day at a recommended dosage of 380 µg/kg once daily. Maralixibat received its first approval on 29 September 2021, from the US Food and Drug Administration (FDA), for use in the treatment of cholestatic pruritus in patients 1 year of age and older with ALGS. Due to its key role in bile acid reabsorption, IBAT was seen an ideal target for pharmacological interruption of bile acid transport. After surgical interruption of enterohepatic circulation was shown to have some success in reducing pruritus in patients with ALGS, pharmacological interruption of bile acid transport was proposed as an alternative to surgical intervention to manage pruritus. Maralixibat (Livmarli™) is an orally-administered, small-molecule ileal bile acid transporter (IBAT) inhibitor being developed by Mirum Pharmaceuticals for the treatment of rare cholestatic liver diseases including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia.
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