- Improvement in pruritus, serum bile acid levels and bilirubin were highly predictive of six-year event-free and transplant-free survival in patients with cholestatic pruritus in Alagille syndrome treated with LIVMARLI
- Analysis highlights for the first-time potential markers associated with better outcomes to help drive clinical care in patients with Alagille syndrome
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that data from LIVMARLI® (maralixibat) oral solution clinical studies evaluating patients with Alagille syndrome (ALGS) were published in Hepatology. LIVMARLI is the first and only approved treatment for cholestatic pruritus in patients with Alagille syndrome three months of age and older.
The overall findings from the retrospective analysis suggest that patients with ALGS treated with LIVMARLI who meet the identified prognostic parameters may have improved six-year survival and clinical outcomes. In the analysis, 46 pre-specified variables were evaluated as potential predictors of event-free and transplant-free survival (EFS and TFS) from the LIVMARLI ALGS clinical program. Patients were assessed for up to six years; EFS was defined as absence of liver transplant (LT), surgical biliary diversion (SBD), hepatic decompensation, or death; TFS was absence of LT or death.
Results from the analysis:
- Improvement in pruritus from baseline to week 48 with LIVMARLI was predictive of six-year EFS and TFS compared to patients with minimal to no improvement in pruritus; 88% vs 57% (p = 0.0046) and 93% vs 57%, (p = 0.0007), respectively.
- Week 48 serum bile acids (sBA) of