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.
“The extensive data being presented at NASPGHAN continues to underscore the impact of LIVMARLI across these pediatric cholestatic indications, and we are excited about what this means for the patient community,” said Pam Vig, PhD, head of research and development at Mirum. “The body of research presented at this conference advances our understanding of LIVMARLI’s clinical effect, which is critical for disease management in the real-world setting.”
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The full presentations can be found on Mirum’s website within the Publications & Presentations section, and each is also linked within the titles below.
Alagille Syndrome (ALGS)
- Abstract 69: Maralixibat for the treatment of severe xanthomas in two children with Alagille syndrome (poster)
- Abstract 71: Maralixibat persistency and adherence for the treatment of cholestatic pruritus in Alagille Syndrome: Real-world experience in the United States (poster)
- Abstract 72: Maralixibat impact on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States (poster)
- Abstract 88: Impact of maralixibat on cholestatic pruritus in young adults aged 16 years and older with Alagille syndrome (poster)
- Abstract 89: Maralixibat, an ileal bile acid transporter inhibitor, delays the need for liver transplant in patients with Alagille syndrome: Real-world experience (poster)
- Abstract 626: Real-world safety experience in patients with Alagille syndrome treated with maralixibat (poster)
- Abstract 632: Maralixibat improves xanthomas and hypercholesterolemia in children with Alagille syndrome: An integrated analysis from two clinical trials (poster)
Progressive Familial Intrahepatic Cholestasis (PFIC)
- Abstract 531: Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH): A randomized placebo-controlled phase 3 study (oral presentation)
**Gerard Odell Prize for Excellence in Pediatric Hepatology Research Recipient** - Abstract 65: Maralixibat improves cholestatic pruritus and bile acids in children with FIC1: Data from the MARCH trial (poster)
- Abstract 85: Long-term maintenance of response and improved liver health with maralixibat in patients with progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH-ON study (poster)
- Abstract 86: Maralixibat leads to significant reductions in bilirubin for patients with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
- Abstract 347: Analysis of long-term safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from MARCH-ON (poster)
- Abstract 625: Analysis of safety in maralixibat-treated participants with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
- Abstract 630: Maralixibat leads to significant reductions in pruritus and improvements in sleep for children with progressive familial intrahepatic cholestasis: Data from the MARCH trial (poster)
SOURCE: BusinessWire