Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome.

Background: Alagille syndrome (ALGS) is a multisystem cholestatic disorder. Maralixibat is approved for the treatment of cholestatic pruritus in ALGS with limited data in adults.

Methods: Participants were included if they received ≥ 2 doses of maralixibat at age ≥ 16 years in one of the three previously published maralixibat ALGS clinical trials.

Results: Eleven initiated treatment < 16 years old (median age, 13.0) with a median follow-up of 4.1 years. Three participants began maralixibat at ≥ 16 years old, with a median follow-up of 3.8 years. Participants starting maralixibat at age < 16 had minimal-to-no itch (change from baseline [CFB]: -1.8; p = 0.002) persisting throughout the study. Serum bile acids (sBA) decreased (CFB: 29 μmol/L; p = 0.03), persisting throughout the study. Participants starting maralixibat ≥ 16 years old had pruritus improvements (CFB: -2.8, -0.6, -1.0). One had a large decrease in sBA (CFB: -112 μmol/L), and two had small increases (CFB: 8, 11 μmol/L). Maralixibat was well tolerated.

Conclusion: Participants receiving maralixibat in adolescence demonstrated improvements in pruritus and sBA, which persisted through young adulthood.