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The Lancet Publishes Data From Mirum Pharmaceuticals' ICONIC Pivotal Study of Maralixibat (LIVMARLI) Treatment in Alagille Syndrome
- Maralixibat data demonstrate four-year durable and clinically meaningful improvements across multiple cholestasis parameters including pruritus. FOSTER
About this update from Mirum Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Maralixibat data demonstrate four-year durable and clinically meaningful improvements across multiple cholestasis parameters including pruritus.\n\n FOSTER CITY, Calif.--(BUSINESS WIRE)--\nMirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leader in rare liver disease, today announced that The Lancet has published four-year data highlighting the safety and efficacy of maralixibat in patients with Alagille syndrome (ALGS), a rare liver disease affecting one in 30,000 children. Maralixibat is an ileal bile acid transporter (IBAT) inhibitor that interrupts enterohepatic bile acid recirculation and has recently been approved by the U.S. FDA under the name of LIVMARLI. Data published in The Lancet demonstrate that maralixibat provides durable and clinically meaningful improvements across multiple disease parameters and has the potential to provide a new treatment paradigm for patients with ALGS.\n\n“Maralixibat is the first medication shown to deliver durable and clinically meaningful improvements in cholestasis for patients treated through the course of the four-year clinical study,” said Professor Emmanuel Gonzales, MD, Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP, Université Paris-Saclay, Le Kremlin-Bicêtre, France and lead author of the publication in The Lancet. “These data are important as they signal a meaningful treatment opportunity to address chronic cholestasis and to potentially extend transplant-free survival for patients with ALGS.”\n\nALGS is a rare, life-threatening multisystem disease that first occurs in childhood with a range of clinical manifestations, including pruritus, jaundice, failure to thrive, xanthomas, and progressive liver disease, which can lead to liver transplantation. In an analysis of children with ALGS conducted by the Global Alagille Alliance (GALA) study, 10 and 18-year native liver survival in 911 ALGS patients presenting with neonatal cholestasis was only 57% and 41%, respectively, representing a previously underappreciated burden of liver disease in ALGS.\n\nThe Lancet publication presents data from the pivotal Phase 2b ICONIC study (n=31) of maralixibat for patients with ALGS. ICONIC demonstrates a significant multi-parameter clinical response including pruritus, serum bile acid (sBA), xanthomas, improved quality of life, and growth. By inhibiting the enterohepatic circulation, maralixibat ...