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Alagille Syndrome
Conference Roundup

Study presented at EASL 2024 shows long-term effectiveness of maralixibat for ALGS

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Long-term efficacy and safety data presented at the European Association for the Study of the Liver (EASL) Congress 2024 demonstrated significant and sustained improvements in liver function among patients with Alagille syndrome (ALGS) treated with maralixibat over an extended period.

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The study, led by Dr Anand Kamath, followed a cohort of patients with ALGS over an extended period, some up to 7 years, documenting their response to maralixibat treatment.

Almost all participants who continued treatment over 7-years experienced notable improvements, such as reduced pruritus and serum bile acid levels. In addition, positive outcomes like improvements in height were observed and maintained throughout the study. Reductions in direct bilirubin were noted among participants on therapy, while there were slight increases in liver transaminases.

Key highlights from the study include:

  • High Long-Term Efficacy: The majority of patients (95% for >3 years, 94% for >5 years, and 88% for >7 years) maintain a persistent response over the long term, indicating the treatment’s durability and effectiveness.
  • Decreasing Trend: While the persistent response rate is high, there is a slight decrease over time (from 95% at >3 years to 88% at >7 years), suggesting that the treatment remains largely effective but may slightly diminish in a small subset of patients over extended periods.
  • Low Intermittent/Loss of Response: The data shows a consistently low percentage of patients experiencing an intermittent or loss of response (5% at >3 years, 6% at >5 years, and 12% at >7 years), which indicates that only a minority of patients may experience a decrease in efficacy or variability in their response over time.

Reference
Kamath A, et al. Clinical benefits with maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: Data from the MERGE study. Poster presented at: European Association for the Study of the Liver (EASL) Congress 2024: June 5-8, 2024.

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