Mirum Pharmaceuticals, Inc. announced the presentation of a series of analyses demonstrating further benefit of LIVMARLI® (maralixibat) oral solution in patients with cholestatic pruritus associated with Alagille syndrome (ALGS) and for patients with progressive familial intrahepatic cholestasis (PFIC), two rare debilitating liver diseases. LIVMARLI is the first medication approved by the U.S. Food and Drug Administration (FDA) to treat cholestatic pruritus in patients with ALGS, and the only to be approved for patients three months of age and older. Data presented during NASPGHAN demonstrated continued evidence of the impact of LIVMARLI for patients with PFIC as well as both clinical data and real-world use of LIVMARLI in patients with ALGS. Presentations included: PFIC: Data from the MARCH PFIC Phase 3 Study was awarded the prestigious Gerard Odell prize for Excellence in Pediatric Hepatology Research. The MARCH PFIC clinical trial was conducted across the broadest range of PFIC types ever studied. Key data from the study demonstrated significant, rapid, and clinically meaningful improvements in pruritus and serum bile acids (sBA) along with significant improvements in bilirubin and weight z-scores. Other key PFIC data included analyses on bilirubin normalization, improvement in sleep, long term maintenance of efficacy from the MARCH-ON extension study, and results from the FIC1 genetic cohort. ALGS: Real-world data in patients with ALGS was presented on adherence to LIVMARLI as well as reduction of other medications and vitamin supplementation while on LIVMARLI. In addition, case studies on delay of liver transplant due to significant reductions in pruritus, and quality of life were shown. Other key ALGS data included clinically meaningful reductions in the number and severity of xanthomas, and improvement in pruritus and maintenance of effect in adult patients with ALGS. LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor and the only approved medication by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older.
LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. LIVMARLI can cause side effects, including: Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment.