Mirum Pharmaceuticals, Inc. announced that the European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients three months of age and older. The approval follows a positive opinion by the CHMP which concluded that LIVMARLI in PFIC brings significant clinical benefit based on improved efficacy and a major contribution to patient care compared to the existing approved treatment for PFIC. Further, evaluation by COMP recommended maintenance of Orphan Drug Designation for LIVMARLI in PFIC.

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. Data from MARCH showed statistically significant reduction in pruritus (p<0.0001) and serum bile acids (p<0.0001) between LIVMARLI versus placebo in the All-PFIC cohort (n=64). Significant improvements were also observed in total bilirubin and growth versus placebo.

The most common treatment emergent adverse event was diarrhea, which was predominantly mild, with no severe cases, and transient. LIVMARLI is also approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with PFIC five years of age and older. Mirum has submitted a supplemental new drug application to introduce a higher concentration formulation of LIVMARLI, used during the MARCH study, to enable label expansion for younger patients with PFIC, and expects to receive FDA feedback 2024.