Maralixibat (Livmarli™) treatment improved event-free survival rates in patients with Alagille syndrome, according to a 6-year real-world evidence analysis. This analysis provides a method for analyzing the outcome of therapeutic interventions in the long term, where comparisons with placebos aren’t feasible. 

These results come from a comparison between the Global ALagille Alliance (GALA) clinical research database and a maralixibat database. The GALA is currently the largest database containing clinical, genetic, and laboratory data on about 1438 patients with Alagille syndrome, and the maralixibat database contains data from up to 6 years on 84 Alagille syndrome patients treated with the drug.

After following the filtration to align key maralixibat eligibility criteria, 469 patients from the GALA were included in the current study. There were no statistically significant differences between patients treated with maralixibat and patients from the GALA in terms of age, mutation, region, or levels of total bilirubin, gamma-glutamyl transferase, and alanine aminotransferase.

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However, the median serum bile acids levels at baseline were significantly higher in the group treated with maralixibat. The rate of event-free survival was significantly higher in the maralixibat-treated group compared to the rate in the GALA database (75% vs 50%).

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Limitations included having no standardized measure of pruritus, limited data availability for serum bile-acid in GALA, as well as an inherent bias for patients who enter a clinical trial. “Limitations will always be present given lack of prospective conduct and inherent biases,” the authors wrote. “Though sensitivity analyses can help mitigate and aid interpretation.”

Event-free survival is defined as the time to a first liver event such as variceal bleeding or ascites requiring therapy, surgical biliary diversion, liver transplantation, or death.

Maralixibat is an ileal bile acid transporter inhibitor by Mirum Pharmaceuticals. The US Food and Drug Administration (FDA) approved maralixibat to treat cholestatic pruritus in Alagille syndrome patients, aged 1 year and older.

These results will be presented at the American Association for the Study of Liver Diseases annual meeting on Monday, November 15, 2021, at 12:30 PM Eastern time by Bettina Hansen, MSc, PhD, scientific committee member of GALA.


Hansen BE, Vandriel SM, Vig P, et al. Application of real-world evidence analytics: a 6-year event-free survival analysis in Alagille syndrome of the GALA clinical research database and maralixibat treated patients. Presented at: The Liver Meeting 2021: November 12-15, 2021; Virtual.

New LIVMARLI™ (maralixibat) data to be showcased at AASLD The Liver Meeting® 2021. News release. Mirum Pharmaceuticals; November 1, 2021.