Long-term maralixibat use benefits adolescents with Alagille syndrome

Maralixibat provides sustained improvements in pruritus and serum bile acids for adolescents with Alagille syndrome, with benefits persisting into young adulthood, according to a new study.

The study included participants from previous maralixibat ALGS clinical trials who received at least 2 doses at age 16 or older. Of the 14 patients, 11 initiated treatment before age 16 (median age, 13), with a median follow-up of 4.1 years. Three started at 16 or older, with a median follow-up of 3.8 years.

Patients who began treatment before age 16 experienced minimal-to-no itch (change from baseline [CFB]: -1.8; P = 0.002) and sustained reductions in sBA (CFB: -29 μmol/L; P = 0.03). Those who started at 16 or older also showed improvements in pruritus (CFB: -2.8, -0.6, -1.0), though sBA responses varied. One participant had a substantial decrease (-112 μmol/L), while 2 had minor increases (8, 11 μmol/L).

Maralixibat was well tolerated by all participants, reinforcing its potential long-term benefit for managing cholestatic pruritus in ALGS from adolescence into young adulthood.

Reference
Hirschfield G, Vandriel SM, Mogul DB, et al. Maralixibat Reduces Serum Bile Acids and Improves Cholestatic Pruritus in Adolescents With Alagille Syndrome. Liver Int. 2025;45(2):e16201. doi: 10.1111/liv.16201. PMID: 39823161; PMCID: PMC11740003.