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Mirum Submits for European Approval of LIVMARLI in Progressive Familial Intrahepatic Cholestasis
Marketing Authorization variation submitted for treatment of patients with progressive familial intrahepatic cholestasis (PFIC) two months of age and older;
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nMarketing Authorization variation submitted for treatment of patients with progressive familial intrahepatic cholestasis (PFIC) two months of age and older; data supports treatment across the broadest range of genetic types ever studied.\n\n\n\n\n\n\nApplication based on statistically significant and clinically meaningful improvements in serum bile acids, pruritus, bilirubin, and growth from the Phase 3 MARCH study.\n\n\n\n\n\n\nSupplemental new drug application for LIVMARLI previously submitted to U.S. FDA for treatment of cholestatic pruritus in patients with PFIC.\n\n\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today 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.\n\n\nThe 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","length":1939,"tagName":"div"}]