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The first! FDA approves innovative treatments for liver disease in children

 Last Update: 2021-10-10
 Source: Internet
 Author: User

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Mirum Pharmaceuticals announced today that the US FDA has approved Livmarli (maralixibat) for the treatment of cholestatic pruritus in children with Alagille syndrome (ALGS) over 1 year old

Alagille syndrome is a rare genetic disease.

Livmarli is an oral selective apical sodium-dependent bile acid transporter (ASBT) inhibitor.

▲The mechanism of action of Livmarli (picture source: Mirum's official website)

The approval of Livmarli is based on the results of a pivotal Phase 2b clinical trial.

Reference materials:

[1] US FDA Approves LIVMARLI (maralixibat) as the First and Only Approved Medication for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome One Year of Age and Older.

[2] Phase 2 placebo-controlled withdrawal study of the ASBT inhibitor maralixibat in children with Alagille syndrome, Retrieved October 28, 2019, from https://mirumpharma.

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