Mirum Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved LIVMARLI®? (maralixibat) oral solution for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis (PFIC). Mirum has also submitted an additional supplemental new drug application (sNDA) to introduce a higher concentration formulation of LIVMARLI, used during the MARCH study, to enable label expansion for younger patients with PFIC, later this year.

LIVMARLI is also approved for the treatment of cholESTatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. (three months and older), Europe (two months and older), in Canada and other regions globally. The approval is based on data from the Phase 3 MARCH study, the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status.