Mirum Pharmaceuticals has completed enrolment in the Phase III EXPAND study assessing Livmarli (maralixibat) for patients aged six months and above with cholestatic pruritus linked to rare cholestatic liver diseases, including biliary atresia.
Cholestatic liver diseases are associated with impaired bile flow, increased serum bile acids, severe itch (pruritus), and reduced quality of life.
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The double-blind, randomised, placebo-controlled trial will assess the efficacy and safety of Livmarli in both children and adults.
Livmarli is an orally administered ileal bile acid transporter (IBAT) inhibitor currently approved by the US Food and Drug Administration (FDA) for paediatric patients with Alagille syndrome and PFIC.
While regulatory approvals for Livmarli exist in the European Union and the US for Alagille syndrome and progressive familial intrahepatic cholestasis (PFIC), patients with other rare forms of these diseases continue to have limited therapeutic options.
The EXPAND study’s main objective is to track changes in pruritus severity from baseline through week 20, with secondary endpoints including alterations in serum bile acids and additional markers of cholestatic liver disease.
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By GlobalDataThe study enrolled patients with a variety of rare conditions, notably including biliary atresia, for which Livmarli is not yet approved.
Patients in the 20-week controlled period are randomised to receive either maralixibat 285µg/kg twice daily or placebo.
Mirum Pharmaceuticals’ chief medical officer Joanne Quan said: “Completing enrolment in EXPAND marks an important milestone in our efforts to broaden the reach of Livmarli to additional patients living with cholestatic pruritus.
“Itch due to cholestasis profoundly affects the daily lives of patients and their families. We aim to address the unmet medical needs of these patients by leveraging our established IBAT mechanism.”
Top line data from the EXPAND study are expected in the fourth quarter of 2026.
