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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.

Reference
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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