mp.weixin.qq.com· 罕见病信息网·· 7 hours agoScore63
Maralixibat Posts Positive EXPAND Results in Ultra-Rare Cholestatic Liver Disease Including Biliary Atresia
III期研究成功,拟于2027年上半年递交补充上市申请
In brief
LIVMARLI (maralixibat) delivered positive topline results in the Phase III EXPAND study, a randomized, double-blind, placebo-controlled trial enrolling 52 patients with ultra-rare cholestatic liver diseases including biliary atresia: 46 children and 6 adults, all aged 6 months and older with cholestatic pruritus. Mirum Pharmaceuticals plans to submit a supplemental marketing application in the first half of 2027.
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