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Mirum’s Maralixibat Demonstrates Transplant-Free Survival for Pediatric Patients with Progressive Familial Intrahepatic Cholestasis
- Maralixibat-treated patients achieving serum bile acid control have five-year native liver survival after treatment. - Data demonstrate normalization and
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Maralixibat-treated patients achieving serum bile acid control have five-year native liver survival after treatment.\n\n\n- Data demonstrate normalization and reductions of liver enzymes, reduced pruritus, and improved growth.\n\n\n- Data selected for oral presentation at the Digital International Liver Congress 2020 being held August 27-29, 2020.\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today reported an analysis from its Phase 2 INDIGO open-label study evaluating maralixibat in patients with bile acid salt export pump (BSEP) deficiency, or PFIC2. The data demonstrated that patients with long-term maralixibat treatment who achieved serum bile acid (sBA) control, have five-year transplant-free survival after treatment.\n\n\n“Patients need alternatives to surgery or transplant to treat PFIC2, otherwise known as BSEP deficiency, and the long-term maralixibat data demonstrate the therapy’s potential to provide an efficacious and well-tolerated treatment for this condition,” said Richard Thompson, Professor of Molecular Hepatology at King’s College London. “The long-term control of serum bile acids underscores the potential for maralixibat to transform the way PFIC2 is treated, as well as provide additional benefit across many of the quality of life measures that can be debilitating and significantly impact patients’ lives.”\n\n\nA study recently published by the NAPPED Consortium established that sBA control (","length":1909,"tagName":"div"}]