Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) has announced the successful results of its Phase 3 EXPAND study, which evaluated LIVMARLI® (maralixibat) for treating cholestatic pruritus in patients aged six months or older with ultra-rare cholestatic liver diseases, including biliary atresia.
The study achieved its primary endpoint and showed statistical significance across all five prespecified secondary endpoints, particularly in patients with biliary atresia and a native liver.
A total of 52 patients participated in the study, comprising 46 pediatric and six adult patients, who were randomized to receive either maralixibat 285 µg/kg twice daily or a placebo over a 20-week double-blind period.
The treatment with maralixibat resulted in a statistically significant improvement in pruritus among the primary analysis population of pediatric patients compared to the placebo group.
Safety findings from the EXPAND study were consistent with previous clinical experiences with maralixibat, with no new safety concerns identified.
Mirum Pharmaceuticals plans to submit a supplemental New Drug Application in the United States in the first half of 2027 based on these results.
Rob Myers, M.D., Chief Medical Officer, stated, “These results are especially important because patients with ultra-rare cholestatic liver diseases continue to face a significant burden from pruritus."