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Mirum’s Long-Term Alagille Data Support Case For Early Filing

 
• By Joseph Haas

Newly public Mirum hopes to file maralixibat for US approval in pruritus due to Alagille syndrome based on Phase IIb data, including long-term extension data reported at AASLD.

Liver Meeting Update, Boston 2019
Mirum is advancing former Shire compounds in cholestatic liver disease

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.

At the American Association for the Study of Liver Diseases meeting earlier this month, Mirum reported that maralixibat has demonstrated...

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