FDA approves Mirum Pharma's first treatment for rare pediatric bile duct disease
The FDA on Wednesday approved Mirum Pharmaceuticals’ first drug, Livmarli (maralixibat), which is also the first treatment for life-threatening cholestatic pruritus in young patients with the super rare Alagille syndrome (ALGS).
ALGS is caused by abnormalities in bile ducts and can lead to progressive liver disease, transplants and potentially death, according to Mirum. An IBAT inhibitor, Livmarli works by blocking bile acid absorption in the small intestines, leading to lower levels of circulating bile acids otherwise linked to severe symptoms, including a severe and unrelenting itch.
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