Data from the pivotal phase 2b ICONIC study of maralixibat in the treatment of Allagile syndrome (ALGS), which indicated durable and clinically meaningful improvements, were recently published in The Lancet.

“Maralixibat is the first medication shown to deliver durable and clinically meaningful improvements in cholestasis for patients treated through the course of the four-year clinical study,” said Emmanuel Gonzales, MD, a professor in France and lead author of the study.

ALGS patients in the study (NCT02160782) treated with maralixibat (Livmarli™) showed clinical improvements in serum bile acid (sBA), pruritus, xanthomas, growth, and quality of life.

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Most (84%) participants showed a reduction of at least 20% in sBA level at week 48. Moreover, improved pruritus response was observed in >80% of participants during the core 48-week period. Treatment effects on sBA level and pruritus were maintained in patients who continued to week 204.

Patients with xanthomas at baseline presented with improved Clinical Xanthoma Scale (CXS) scores at weeks 48 and 204. Also, none of the participants developed new xanthomas while being treated with maralixibat.

Participants’ quality of life and fatigue improved from baseline to week 48. Improvements in fatigue were maintained throughout the 204-week period. Mean height Z score increased from baseline to week 204.

Maralixibat treatment was well-tolerated, with most adverse events being mild to moderate and transient in nature. The most common adverse events were diarrhea and abdominal pain.

“These data are important as they signal a meaningful treatment opportunity to address chronic cholestasis and to potentially extend transplant-free survival for patients with ALGS,” Dr. Gonzales said.


The Lancet publishes data from Mirum Pharmaceuticals’ ICONIC pivotal study of maralixibat (LIVMARLI) treatment in Alagille syndrome. News release. Mirum Pharmaceuticals; November 1, 2021.

Gonzales E, Hardikar W, Stormon M, et al. Efficacy and safety of maralixibat treatment in patients with Alagille syndrome and cholestatic pruritus (ICONIC): a randomised phase 2 study. Lancet. 2021;398(10311):1581-1592. doi:10.1016/S0140-6736(21)01256-3