The relentless itch of rare childhood liver disease just met its match in a landmark trial

Some children are born with rare liver diseases — like biliary atresia, where the bile ducts that drain the liver never formed properly — and one of the cruelest symptoms is itching so intense it can steal a child's sleep night after night. Mirum's medicine LIVMARLI (maralixibat) was tested in 52 patients, most of them children as young as six months, and it met every goal of the Phase 3 trial: itching scores improved significantly versus placebo, and bile acids in the blood — a key marker of the disease — fell 70%. No new safety concerns appeared. The company plans to file for approval in the first half of 2027, which could bring a first real option to families who have had none.