Alagille Syndrome
Conference Roundup

Maralixibat well tolerated in infants <1 year old with ALGS

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Maralixibat was found to be well tolerated in infants < 1 year old with Alagille syndrome (ALGS) with body weight of ≥2.5 kg, according to a poster presented at The Liver Meeting of the American Association for the Study of Liver Diseases.

Use of maralixibat in children with cholestasis 1 year of age and older is already well established and approved for the treatment of cholestatic pruritus in patients with ALGS 1 year of age and older. The current data aimed to characterize the safety and tolerability of maralixibat in children as young as 2 months of age.

In an interim analysis of an ALGS cohort from an ongoing, open-label, Phase 2 trial, 8 infants with ALGS and cholestasis who were <1 year of age with a body weight ≥2.5 kg and gestational age ≥36 weeks were treated with maralixibat (380 µg/kg once daily) in addition to standard-of-care. Exclusion criteria included decompensated cirrhosis.

Patients were treated for a median of 136.5 days. Overall, 87.5% of patients (n = 7) experienced ≥1 treatment-emergent adverse event (TEAEs); most were Grade 1 in severity and unrelated to maralixibat. TEAEs-related to maralixibat included 2 cases of Grade 1 diarrhea.

Half of the patients (n = 4) experienced 7 serious adverse events unrelated to maralixibat; all cases were resolved and a dose change was not required.

Overall, TEAEs were found to be self-limiting and did not interrupt doses, with infections and gastrointestinal disorders the most frequently reported TEAEs.

Gonzales, et al. Safety and tolerability characterization of maralixibat in infants with ALGS from 2 months of age: Interim results from the RISE study. Poster presented at: AASLD 2022.


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