Maralixibat treatment leads to height and weight catch-up in patients with Alagille syndrome (ALGS), according to a new study presented at the European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN) 54th annual meeting. “Patients with the greatest height disadvantage at baseline had the greatest catch-up in height,” the authors of the study said.

Maralixibat treatment is associated with improved event-free survival in ALGS, according to new research. This suggests that the treatment not only improves pruritus, it may also have additional benefits for the liver.

Here, a team of researchers led by Pamela Vig, PhD, the head of research and development at Mirum Pharmaceuticals, evaluated the impact of long-term treatment with the drug on patients’ nutritional status. The researchers analyzed data from 3 clinical trials. They evaluated the height and weight Z-scores of 34 patients at baseline and at week 204.

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They found that the mean height Z-score of the patients went from -1.66 to 1.29 at week 204. The greatest catch-up height gain occurred in patients with the lowest quartile height Z-scores at baseline. Similarly, those with lower weight Z-scores at baseline had greater catch-up weight gain. There was a significant correlation between change in weight and height and baseline weight and height.

Height Z-score increased significantly from baseline at week 204 in patients who had serum bile acid levels less than 200 mmol/L at week 48, but not for those whose serum bile levels were higher than >200 mmol/L. The researchers also found that there was no change in the levels of vitamin D or albumin during the treatment period.

Maralixibat is an ileal bile acid transporter inhibitor that has recently been approved by the US Food and Drug Administration to treat patients with ALGS 1 year and older. 


Kamath BM, Mogul DB, Baek M, Nunes T, Vig P. Maralixibat improves growth in patients with Alagille syndrome: a 4-year analysis. Presented at: the European Society for Paediatric Gastroenterology Hepatology and Nutrition (ESPGHAN) 54th annual meeting: Copenhagen, Denmark, and virtual; June 22-25, 2022.