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Alagille Syndrome
Conference Roundup

Treatment-emergent gastrointestinal effects after maralixibat often mild in ALGS

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Most patients with Alagille syndrome (ALGS) treated with the apical sodium-dependent bile acid transport (ASBT) inhibitor, maralixibat (MRX; Mirum Pharmaceuticals), who experience treatment-emergent gastrointestinal effects do not discontinue treatment, according to study results presented at the 6th World Congress of Paediatric Gastroenterology, Hepatology and Nutrition.

In this analysis, an integrated population of 86 patients with ALGS treated with MRX (66.5 µg/kg to 380 µg/kg once daily up to 380 µg/kg twice daily), were included.

Overall, 56% (n = 48) and 53% (n =46) of patients experienced an event including diarrhea and abdominal pain, respectively. Both diarrhea and abdominal pain were noted in 38% (n = 33) of patients with 23% (n = 20) experiencing the events concurrently.

During the first month of treatment incidence was highest and then decreased over time. Most events were mild to moderate in severity. One patient reported dose interruption due to diarrhea and 1 patient due to adnominal pain. No patients discontinued treatment.

A subgroup analysis showed a similar incidence of gastrointestinal events between patients treated with MRX and placebo.

Reference
Raman R, et al.Gastrointestinal tolerability of maralixibat in patients with Alagille syndrome: An integrated analysis of short- and long-term treatment. Presented at: 6th World Congress of Paediatric Gastroenterology, Hepatology and Nutrition.

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