Mirum Pharmaceuticals (Nasdaq: MIRM), a leader in rare liver disease, says that the US Food and Drug Administration has approved Livmarli (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older.
Livmarli, a minimally absorbed ileal bile acid transporter (IBAT) inhibitor, is the first and only FDA-approved medication in this rare liver disease which affects 2,000 to 2,500 children in the USA. Livmarli is now available for prescribing. In conjunction with the approval, Mirum received a rare pediatric disease priority review voucher.
Mirum priced the drug, to be made available for distribution immediately, at $1,550 per unit, or annual cost at about $391,000 for a 17-kilogram child, the average weight of patients in its studies.
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