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Mirum Pharmaceuticals Presents Maralixibat Five-Year Transplant-Free Survival Data for Patients With PFIC2 at Digital International Liver Congress 2020
Maralixibat demonstrates significant transplant-free survival for patients with progressive familial intrahepatic cholestasis (PFIC2) who achieved serum bile
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n\nMaralixibat demonstrates significant transplant-free survival for patients with progressive familial intrahepatic cholestasis (PFIC2) who achieved serum bile acid (sBA) control in five-year analysis (p=0.0006).\n\n\nsBA responders remain on maralixibat for more than five years with improvements across multiple parameters including normalization of liver enzyme and bilirubin levels, decreased pruritus and improvement in growth.\n\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, presented an analysis from its Phase 2 INDIGO study in an oral late-breaker session at the Digital International Liver Congress™ 2020. The five-year analysis showed that patients with PFIC2, also known as bile salt export pump (BSEP) deficiency, who achieved sBA control on long-term maralixibat treatment have a significant improvement in transplant-free survival.\nThis press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200829005005/en/(Graphic: Business Wire)\n\n“The maralixibat data demonstrate a well-tolerated, long-term treatment option to address PFIC2, which may provide patients with not only a viable treatment alternative to liver transplantation, but also a possible path to addressing many of the quality of life challenges that can make PFIC a burdensome disease for patients and their families,” said Richard Thompson, professor of molecular hepatology at King’s College London, who presented the data.\n\n\nLate-breaker oral presentation\n\n\nAugust 29, 2020 – 2:45-3:00pm CET\nLB008: Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency.\n\n\nIn February 2020, the NAPPED consortium published an analysis showing that surgical interruption of the enterohepatic circulation of bile acids can lead to transplant-free survival when sBAs are reduced by 75% or below 102 µmol/L. Data presented today demonstrate that, similar to the findings of NAPPED, patients exhibiting sBA control with pharmacological interruption of the enterohepatic circulation with maralixibat have significantly imp...