Mirum ex­pands la­bel for rare liv­er dis­ease drug, serv­ing up more com­pe­ti­tion for Ipsen's Byl­vay

The FDA on Wednes­day ex­pand­ed the ap­proval of Mirum Phar­ma­ceu­ti­cals’ Liv­mar­li (mar­al­ix­i­bat) for the treat­ment of in­tense itch­ing re­lat­ed to a rare liv­er dis­ease in adults and chil­dren 5 years and old­er known as pro­gres­sive fa­mil­ial in­tra­hep­at­ic cholesta­sis (PF­IC).

Liv­mar­li, an ileal bile acid trans­porter (IBAT) in­hibitor, pre­vi­ous­ly won ap­proval in 2021 as an itch re­lief treat­ment for chil­dren 3 months and old­er with the ge­net­ic dis­or­der known as Alag­ille syn­drome. The new in­di­ca­tion’s ap­proval is based on the largest-ever Phase III tri­al in PF­IC, with 93 pa­tients across a range of ge­net­ic types.

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