FDA approves Livmarli (maralixibat) for the treatment of cholestatic pruritus in patients five years of age and older with progressive familial intrahepatic cholestasis
Mirum Pharmaceuticals, Inc. announced that the 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.
“Livmarli has the potential to have a transformational impact for patients with cholestatic pruritus associated with PFIC, and importantly, offers an option for those patients with the rarest of subtypes,” said Chris Peetz, chief executive officer at Mirum. “Thank you to the patients, families, and clinicians whose study participation made this approval possible.”
The approval is based on data from the Phase III 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.
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