MIRM - Mirum files for expanded approval of Livmarli in US

• Mirum Pharmaceuticals ( NASDAQ: MIRM ) submitted a supplemental new drug application (sNDA) to the U.S. Food and Drug Administration (FDA) seeking expanded approval of Livmarli oral solution to treat cholestatic pruritus (itch) in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC).
• PFIC is a disorder which causes progressive liver disease, which usually leads to liver failure. In patients with PFIC, liver cells are not able to secrete enough bile, and this buildup of bile leads to liver disease.
• Livmarli (maralixibat) is already approved in the U.S. and Israel to treat cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, and in the EU for the same use in patients two months of age and older. ALGS is an inherited disorder in which bile builds up in the liver due to fewer bile ducts to drain the bile.
• The sNDA was backed by data from a phase 3 trial called MARCH PFIC.
• MIRM +1.10% to 23.80 premarket Feb. 14

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