Mirum Snags First Drug Approval for Rare, Genetic Liver Disorder

Mirum Pharmaceuticals has received approval from the U.S. Food and Drug Administration (FDA) to start selling its oral medication LIVMARLI to treat cholestatic pruritus in patients diagnosed with Alagille syndrome (ALGS). 

LIVMARLI (maralixibat) is indicated for ages one year and older. It is an orally administered, once-daily ileal bile acid transporter (IBAT) inhibitor. It will now be accessible to patients with a prescription through Mirum Access Plus, the company’s patient support services program and single-source specialty pharmacy. The MAP program works with healthcare providers and families to provide access support, insurance coverage, and financial support options for the drug. 

ALGS is a rare genetic disease caused by bile duct abnormalities, leading to progressive liver disease. The shrunken or malformed bile ducts cause cholestasis, which eventually develops into inflammation and liver injury. It affects 2,000 to 2,5000 children in the U.S. alone. The estimated incidence of ALGS is one in every 30,000 people. 

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