Business
Mirum Pharmaceuticals Provides Corporate Update
- Completion of maralixibat rolling NDA targeted for Q1 2021 - Expecting first-patient-in for volixibat studies in ICP and PSC, and maralixibat study in
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Completion of maralixibat rolling NDA targeted for Q1 2021\n\n- Expecting first-patient-in for volixibat studies in ICP and PSC, and maralixibat study in biliary atresia in Q1 2021\n\n- Launching volixibat primary biliary cholangitis program in H2 2021\n\n- European commercial leader hired in anticipation of potential approval of maralixibat\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases, today provided a corporate update in advance of the company’s presentation at the 39th Annual J.P. Morgan Healthcare Conference.\n\n“I am very excited about what 2021 holds for Mirum. In the first quarter alone, we expect to complete our rolling NDA submission for maralixibat, taking us one step closer to making this medicine available to patients with Alagille syndrome, and expect to initiate three randomized studies of maralixibat and volixibat in new indications,” said Chris Peetz, president and chief executive officer at Mirum. “Today we are announcing key progress in maralixibat launch readiness and further expansion of the volixibat program into primary biliary cholangitis with an additional, potentially registrational, randomized study.”\n\n2021 Pipeline Highlights\n\n- Expects completion of rolling new drug application (NDA) submission to the U.S. Food and Drug Administration (FDA) for maralixibat for the treatment of cholestatic pruritus associated with Alagille syndrome in the first quarter of 2021.\n\n- Ongoing review of the marketing authorization application to the European Medicines Agency for maralixibat in progressive familial intrahepatic cholestasis, type 2 (PFIC2) in Europe.\n\n- Anticipates randomizing patients into three clinical studies in the first quarter of 2021:\n\n\nPhase 2a/2b study (OHANA) of volixibat for the treatment of intrahepatic cholestasis of pregnancy.\n\n\nPhase 2b study (VISTAS) of volixibat for the treatment of pruritus associated with primary sclerosing cholangitis.\n\n\nPhase 2 study (EMBARK) of maralixibat for the treatment of biliary atresia.\n\n\n- Planning to initiate a Phase 2b study of volixibat for the treatment of pruritus associated with primary biliary cholangitis in the second half of 2021.\n\nCor...