Livmarli filed with FDA for sNDA in cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis
Mirum Pharmaceuticals announced that it has submitted a supplemental New Drug Application (sNDA) for Livmarli (maralixibat) oral solution for the treatment of cholestatic pruritus in patients two months of age and older with progressive familial intrahepatic cholestasis (PFIC)
The sNDA submission is based on data from the MARCH PFIC Phase III study of Livmarli. MARCH PFIC is the largest randomized trial conducted in PFIC, with 93 patients across a range of genetic PFIC subtypes, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status.
In the study, Livmarli-treated patients had statistically significant improvements in pruritus (p< 0.0001), serum bile acids (p<0.0001), bilirubin (p="0.0471)," and growth as measured by weight z-score (p="0.0391)," in the cohort evaluating combined genetic subtypes. the snda also includes data from the phase ii indigo study of pfic2 patients demonstrating transplant-free survival in all serum bile acid responders after more than five years of treatment with livmarli.
Livmarli is currently approved in the U.S. and Israel for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older, as well as in the European Union in the same indication, in patients two months of age and older.
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