Alagille Syndrome
Conference Roundup

Maralixibat well tolerated in patients with ALGS in real-world setting

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Patients with ALGS treated with maralixibat in a real-world analysis had lower rates of adverse events (AEs) than described in clinical trials, according to data presented at The Liver Meeting of the American Association for the Study of Liver Diseases.

Real-world safety data from all patients treated with maralixibat through the initial Expanded Access Program treated with a target dose of 380 µg/kg daily was analyzed.

The baseline median age was 6 years, median height was 103cm, and median weight was 16 kg. Patients were followed for a median of 243 days.

Most treatment-emergent adverse events (TEAEs) were considered mild and not related to maralixibat, with 8.1% of patients experiencing gastrointestinal TEAEs, which were mild and did not lead to dose interruptions or treatment discontinuations.

One patient discontinued treatment due to transaminase elevation (grade 3) of unknown clinical significance.

Himes RW, et al. Real-world safety experience in patients with Alagille syndrome treated with maralixibat. Poster presented at: AASLD 2022.

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