Mirum Pharmaceuticals: EU approves LIVMARLI for patients with PFIC

Mirum Pharmaceuticals announced that the European Commission has granted marketing authorization for LIVMARLI or maralixibat, oral solution for the treatment of progressive familial intrahepatic cholestasis or 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 patient community in Europe will greatly benefit from LIVMARLI’s approval, supported by years of impressive data showing improvements in the most burdensome aspects of disease,” said Emily Ventura, executive director, PFIC Network. “PFIC can be life-altering and can have a devastating impact on patients. We are encouraged to see that young patients will have a new medication and hope for a life less burdened by cholestasis.” 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 this year.