Mirum Pharmaceuticals, Inc. announced submission to the European Medicines Agency (EMA) of a variation application to the Marketing Authorization for LIVMARLI (maralixibat) oral solution, to extend the label for progressive familial intrahepatic cholestasis (PFIC) in patients two months of age and older. LIVMARLI is currently approved by the EMA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older.
The submission is based on data from the Phase 3 MARCH study of LIVMARLI in patients with PFIC. MARCH is the largest randomized trial conducted in PFIC, with 93 patients across a broad range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status. In the cohort evaluating combined genetic types, 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). The variation also included interim analysis data from the ongoing open-label extension study (MARCH-ON) which included 85 of the 93 patients that were enrolled in the pivotal trial.
“The expansive data we have collected from our PFIC studies give us great confidence that, if approved by the EMA, LIVMARLI will provide an impactful treatment option for patients across a broad range of genetic types, including those with phenotypic diagnosis but without a known variant,” said Chris Peetz, president and chief executive officer at Mirum. “We have now submitted these groundbreaking data to regulatory agencies in the U.S. and Europe with the hope of making LIVMARLI available to PFIC patients across multiple geographies. We are grateful to the clinicians and families who participated in the research that has meaningfully advanced treatment for PFIC.”
LIVMARLI is currently approved in the United States and Europe for the treatment of cholestatic pruritus in patients with Alagille syndrome three months of age and older, and two months of age and older, respectively. In addition to the Marketing Authorization variation announced today, Mirum has also submitted a supplemental new drug application to the U.S. FDA for the treatment of cholestatic pruritus in patients three months of age and older with PFIC.
SOURCE: Businesswire