Mirum Pharmaceuticals presented new data showing that maralixibat (LIVMARLI®) oral solution is safe and well tolerated in patients with Alagille syndrome (ALGS) as young as 2 months of age.

The results were presented at the 2022 The Liver Meeting of the American Association for the Study of Liver Diseases (AASLD).

“Interim safety data in ALGS underscores LIVMARLI’s safety and tolerability profile in infants as young as 2 months of age. We are thrilled to expand the growing body of clinical evidence for LIVMARLI in both PFIC [progressive familial intrahepatic cholestasis] and ALGS and to present our late breaking data at the AASLD conference,” said Pam Vig, PhD, head of R&D at Mirum Pharmaceuticals.

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The treatment-emergent adverse events (TEAEs) reported during the RISE study were mostly mild in severity (grade 1) and unrelated to maralixibat. Seven serious adverse events unrelated to maralixibat were reported in 4 participants but none required a change in maralixibat dose or treatment discontinuation.

The RISE study enrolled 8 patients younger than 1 year of age (average age, 7 months) diagnosed with ALGS or PFIC who received 380 µg/kg of maralixibat once daily. Treatment safety was measured as TEAEs and change from baseline to week 13.

Read more about ALGS diagnosis

In addition, real-world data showed that TEAEs were mostly mild in severity and unrelated to maralixibat, corroborating the results from the RISE study. Moreover, treatment-related gastrointestinal adverse events were observed in 8.1% of study participants. One patient discontinued the treatment due to transaminase elevation (grade 3) of unknown clinical significance potentially related to maralixibat.

Real-world data derived from the analysis of 37 patients with ALGS enrolled in the LIVMARLI global expanded access program. All patients received 380 µg/kg of maralixibat.

Mirum Pharmaceuticals has also presented efficacy and safety data of maralixibat in patients with PFIC from the randomized placebo-controlled MARCH-PFIC trial. This oral session was nominated for Best of Liver Meeting.


Mirum presents new data from LIVMARLI phase 3 PFIC study and ALGS infant study in late-breaker presentations at The Liver Meeting of AASLD. News release. Mirum Pharmaceuticals, Inc.; November 7, 2022.