The Lancet Publishes Data From Mirum Pharmaceuticals' ICONIC Pivotal Study of Maralixibat Treatment in Alagille Syndrome
November 01, 2021 at 06:00 pm IST
Mirum Pharmaceuticals, Inc. 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.