Six-year natural history comparison shows maralixibat improves event-free survival in ALGS

Maralixibat (MRX) may improve event-free survival (EFS) in patients with Alagille syndrome (ALGS) compared to patients with natural history, according to data from a 6-year real-world evidence (RWE) analysis presented at the American Association for the Study of Liver Diseases annual congress, The Liver Meeting.

A total of 84 patients with ≥6 years of data from the MRX database and 469 patients from GALA, the largest global clinical research database for AGLS, were included. Age, total bilirubin (TB), GGT, and ALT were balanced between groups.

The median baseline serum bile acid (sBA) was significantly higher than in the MRX cohort than the GALA group, although 85% of patients in the GALA group did not have sBA data available.

Crude 6-year EFS, defined as the time to first event of hepatic decompensation; surgical biliary diversion; liver transplantation (LT); or death, was significantly better in the MRX cohort than the GALA group (73% vs 50%).

Reference
Hansen B, 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.