Maralixibat

Maralixibat (also known as SHP625, LUM001, and lopixibat) is an ileal bile acid transporter inhibitor, like odevixibat. Maralixibat is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome.

Previously, patients with cholestatic pruritus associated with Alagille syndrome were treated with antihistamines, rifampin, ursodeoxycholic acid, cholestyramine, naltrexone, and sertraline alone or in combination. No clinical trials have been performed to assess the efficacy of these treatments for cholestatic pruritus and treatments were given based on a prescriber's clinical experience. Surgical interventions such as partial external bile diversion and ileal exclusion have also been used as treatments.

Maralixibat represents the first FDA-approved treatment for cholestatic pruritus in patients with Alagille syndrome. It was granted FDA approval on 29 September 2021. In October 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended maralixibat be granted marketing authorization for the treatment of cholestatic pruritus in patients with Alagille syndrome: it was granted marketing authorization in Europe on 13 December 2022. On July 21, 2023, maralixibat was also approved by Health Canada.

Maralixibat is indicated in the treatment of cholestatic pruritus in patients with Alagille syndrome. It is approved for use in patients at least one-month-old in the US and at least two months old in Europe. In Canada, it is reserved for use in adults.