Patients receiving maralixibat treatment for Alagille syndrome (ALGS) may experience gastrointestinal events including abdominal pain and diarrhea, but these symptoms tend to be mild to moderate and transient, and do not lead to discontinuation of treatment.

These findings are being presented as a poster at the North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) Annual Meeting.

The analysis included data from 3 phase 2 clinical trials of maralixibat and encompassed 86 patients. Of those, 49 (57%) experienced diarrhea while 46 (53%) experienced abdominal pain. A total of 33 (38%) patients experienced both diarrhea and abdominal pain and 20 (23%) experienced both concurrently.

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The gastrointestinal events usually appeared within the first month of treatment and decreased over time, according to the poster. They generally lasted for less than a week, were mild to moderate in severity, and did not require changes to maralixibat doses. Only 2 patients had dose interruptions across all 3 trials—1 who experienced abdominal pain and 1 who experienced diarrhea.

No patients discontinued use of maralixibat due to gastrointestinal issues during any of the studies. The studies that also included a placebo-treated group (39 maralixibat and 19 placebo) found similar rates of gastrointestinal events across both groups with slightly more abdominal pain experienced in the treatment group. In those studies, diarrhea was reported in 44% of the treatment group and 50% of the placebo group while abdominal pain was reported in 38% of the treatment group compared to 28% of the placebo group.

“Maralixibat (MRX) is an apical sodium-dependent bile acid transport (ASBT) inhibitor that interrupts enterohepatic recirculation, thus reducing pruritus and cholestasis,” the poster authors wrote.

In the 3 clinical trials, patients received doses of maralixibat from 66.5 μg/kg to 380 μg/kg once daily and up to 380 μg/kg twice daily, depending on the study.


Kamath B, Raman R, Garner W, Tucker E, Vig P, Gonzales E. Gastrointestinal tolerability of maralixibat in patients with Alagillle syndrome: an integrated analysis of short- and long-term treatment. North American Society for Pediatric Gastroenterology, Hepatology, and Nutrition (NASPGHAN) Annual Meeting; December 12-18, 2021; Virtual.