A new study has found that treatment with maralixibat for up to 4 years leads to improved growth in patients with Alagille syndrome (ALGS).

The study, published in Frontline Gastroenterology, noted that patients with the lowest baseline height and weight Z-scores experienced the greatest improvements in these scores.

Maralixibat is an ileal bile acid transporter inhibitor approved by the US Food and Drug Administration (FDA) to treat cholestatic pruritus in patients with ALGS who are at least 3 months of age.

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“Recent data suggest that maralixibat is associated with improved event-free survival in this population, suggesting that the drug may potentially improve liver disease outcomes beyond pruritus control in ALGS,” the authors wrote. “Patients with ALGS clearly have impaired growth and therefore we evaluated the impact of long-term maralixibat treatment on the nutritional status of these patients.”

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The research team assessed data on 34 patients with ALGS who participated in 3 clinical trials of maralixibat and who had height and weight Z-scores available at baseline and at the week 204 follow-up.

After treatment with maralixibat, the mean height Z-score increased from −1.66 to −1.29 at week 204, and the greatest height gain occurred in those with the lowest baseline height Z-scores. There was also a greater weight increase among those with lower baseline weight Z-scores.

Those patients with serum bile acids lower than 200 µmol/L at week 48 achieved an increase in height Z-score from −1.58 at baseline to −1.16 at week 204, whereas those with an serum bile acids higher than 200 µmol/L at week 48 had no significant change in height Z-score. The authors therefore speculate that bile acid homeostasis may be a factor in the improvement of height deficits.

The research team recommends further analyses, including a comparison with a natural history cohort of patients with ALGS, to better understand the impact of treatment with maralixibat on growth in this population.


Kamath B, Mogul D, Baek M, Nunes T, Vig P. Maralixibat improves growth in patients with Alagille syndrome: a 4-year analysis. Frontline Gastroenterol. Published online July 27, 2023. doi:10.1136/flgastro-2023-bspghan.44