Since acquiring a pair of mid-stage apical sodium-dependent bile acid transporter (ASBT) inhibitors from Shire PLC in 2018, Mirum Pharmaceuticals has been working to build a case for both in pediatric cholestatic liver disease. Now, with long-term extension data, the company hopes to convince the US Food and Drug Administration on filing maralixibat for pruritus related to Alagille syndrome on the strength of Phase IIb data.
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