Mirum Reports EXPAND Phase 3 Results for Maralixibat in Rare Liver Diseases
Published in Diseases!, Drugs development & discovery.
Published October 12, 2026.
Mirum Pharmaceuticals announced Phase 3 EXPAND results on October 12 for maralixibat in additional ultra-rare cholestatic liver diseases, including biliary atresia. The randomized, double-blind study included 52 participants: 46 children and six adults.
In the pediatric primary analysis, average worst-daily observer-reported itch scores improved by 1.81 points with maralixibat versus 0.83 with placebo during weeks 13–20. The adjusted difference was 0.98 points (p=0.0004) on the four-point severity range. Serum bile-acid levels also decreased.
Safety: Diarrhea occurred in 64% of treated participants versus 11.1% on placebo. Serious treatment-emergent adverse events occurred in 20% versus 18.5%; the company considered those events unrelated to maralixibat.
Evidence limits: These are company-reported topline findings from a small, 20-week controlled study. They establish neither a cure nor improved transplant-free survival. Although LIVMARLI already has approvals in other cholestatic diseases, EXPAND studied additional settings for which it is not currently approved. Mirum plans a U.S. supplemental application in the first half of 2027.
Source: Mirum announcement via Business Wire, reproduced by MarketScreener, October 12, 2026.