EMA accepts MAA for maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 . Mirum Pharma.

Mirum Pharmaceuticals, Inc., a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, announced that the company’s Marketing Authorization Application (MAA) for its investigational medicine, maralixibat, for the treatment of patients with progressive familial intrahepatic cholestasis type 2 (PFIC2), also known as bile salt export pump (BSEP) deficiency, was accepted for review (validated) by the European Medicines Agency (EMA). The validation of the application by the EMA confirms all essential regulatory elements are included in the submission such that the EMA can begin its review. Data from the Phase II INDIGO study evaluating maralixibat for pediatric patients with PFIC2 served as the basis of the MAA submission. Mirum recently announced data showing five-year transplant-free survival for patients who achieved serum bile acid control. The data also demonstrated improvements across multiple parameters including pruritus control, improvements of liver enzyme and bilirubin levels, and improvement in growth. These data were presented at the annual meeting of the European Association for the Study of the Liver. The MAA submission also includes data on five-year event-free survival with maralixibat compared to the NAPPED natural history cohort. To provide further evidence of maralixibat’s potential in PFIC2 with higher doses and other PFIC subtypes, Mirum is conducting a Phase III study, MARCH, with completion of enrollment expected in the second quarter of 2021.