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Maralixibat is a small molecule, orally administered reversible inhibitor of the ileal bile acid transporter (IBAT), also known as the sodium/bile acid cotransporter. It is primarily used to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). By inhibiting IBAT in the terminal ileum, maralixibat decreases reabsorption of bile acids, leading to increased fecal excretion and reduced systemic bile acid levels. This mechanism helps alleviate pruritus associated with these rare genetic liver diseases. The drug was developed for pediatric and adult populations and is available as an oral solution[1][2][3][4][5][6][8].
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