Business
Mirum Pharmaceuticals Announces European Medicines Agency Validation of the Marketing Authorization Application for Maralixibat in Patients with PFIC2
- Five-year transplant-free survival data from the Phase 2 INDIGO study used as the basis for the MAA submission - Maralixibat would be the first treatment
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Five-year transplant-free survival data from the Phase 2 INDIGO study used as the basis for the MAA submission\n\n- Maralixibat would be the first treatment labeled for patients with PFIC2 in Europe, if approved\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today 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.\n\n“PFIC is life-altering for patients and their families as they struggle to manage the round-the-clock care and surgical decisions that many children often need,” said Chris Peetz, president and chief executive officer at Mirum. “Validating our MAA is a groundbreaking step towards providing a medicine to address PFIC2. Based on the long-term transplant-free survival improvement in maralixibat responders, we believe that maralixibat could provide a treatment alternative to invasive surgeries for these patients, as well as improve quality of life. We are excited about the opportunity to make maralixibat available to patients with PFIC2 in Europe.”\n\nData from the Phase 2 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.\n\nTo provide further evidence of maralixibat’s potential in PFIC2 with higher doses and other P...