Case report demonstrates durable response after treatment with maralixibat in ALGS

Clinical trials of the breakthrough drug, maralixibat, for children aged 3 months and older suffering from Alagille syndrome-related pruritus have extended up to a 4-year follow-up.

A recent report highlights a case in which a patient exhibited not just the anticipated reduction in pruritus but complete resolution of the itching after treatment with maralixibat. The patient’s response demonstrated longevity, persisting for over 7 years, and resulting in the discontinuation of all other antipruritic medications.

Reference
Garcia A, Hsu E, Lin HC. Resolution of Pruritus in a Child With Alagille Syndrome Treated With Maralixibat for Seven Years: Durable Response and Discontinuation of Other Medications. JPGN Rep. 2023;4(3):e335. doi: 10.1097/PG9.0000000000000335. PMID: 37600618; PMCID: PMC10435040.