Mirum's Livmarli hits primary endpoint in Phase 3 biliary atresia study
The EXPAND trial showed a placebo-adjusted 0.98-point improvement in pruritus severity score, with a p-value of 0.0004
Published
Mirum Pharmaceuticals said its Phase 3 EXPAND study of Livmarli (maralixibat) met its primary endpoint in patients with ultra-rare cholestatic liver diseases, including biliary atresia.
The study showed a placebo-adjusted treatment difference of 0.98 points in pruritus severity score, with a p-value of 0.0004. All five prespecified secondary endpoints also reached statistical significance.
Mirum plans to submit a supplemental New Drug Application in the United States in the first half of 2027, a filing window that is still several months away.
The result extends Livmarli's label opportunity beyond its current approved uses and marks Mirum's third late-stage readout this quarter, alongside Atebrioz's approval and the AZURE-1 hepatitis delta data. Unlike the hepatitis delta program, which carries a billion-dollar revenue estimate, the EXPAND results target a narrower pediatric population, making the commercial upside incremental rather than transformative. Even so, the result adds to a pattern of consistent execution across Mirum's pipeline at a time when peers including BridgeBio and Scholar Rock are also advancing rare-disease assets through FDA review.