Mirum Pharmaceuticals Announces Primary Endpoint Met in Phase 3 EXPAND Study of LIVMARLI® (maralixibat) in Ultra-Rare Cholestatic Liver Diseases
Key Highlights
- ➤EXPAND met its primary endpoint, significantly reducing pediatric cholestatic pruritus versus placebo
- ➤Maralixibat cut serum bile acids 69.0% versus placebo; p<0.0001
- ➤Biliary atresia patients with native livers saw significant pruritus reduction; p=0.0002
- ➤Mirum plans U.S. sNDA submission in the first half of 2027
- ➤EXPAND safety findings matched prior experience, with no new safety signals
Expert Statements
Rob Myers, Chief Medical Officer at Mirum Pharmaceuticals
“These results are especially important because patients with ultra-rare cholestatic liver diseases continue to face a significant burden from pruritus.”
Rob Myers, Chief Medical Officer at Mirum Pharmaceuticals
“The improvements in pruritus and reductions in serum bile acids observed in EXPAND reinforce the potential for maralixibat to address this unmet need and benefit a broader population of patients living with ultra-rare cholestatic liver diseases.”
Jennifer Vittorio, Associate Professor of Pediatrics and Medical Director, Pediatric Liver Transplantation, NYU Langone Health
“Cholestatic pruritus can be relentless and profoundly disruptive for people living with ultra-rare cholestatic liver diseases, yet treatment options remain limited.”
Jennifer Vittorio, Associate Professor of Pediatrics and Medical Director, Pediatric Liver Transplantation, NYU Langone Health
“The magnitude and consistency of improvements in pruritus and serum bile acid levels observed in EXPAND are compelling, including for patients with biliary atresia.”
Jen Lau, Co-Founder and Executive Director of BARE, Inc. (Biliary Atresia Research and Education)
“For families living with ultra-rare cholestatic liver diseases, the possibility of new treatment options brings tremendous hope.”
Jen Lau, Co-Founder and Executive Director of BARE, Inc. (Biliary Atresia Research and Education)
“These results are meaningful for the biliary atresia community, where children and families continue to face significant challenges due to cholestatic pruritus.”
Jen Lau, Co-Founder and Executive Director of BARE, Inc. (Biliary Atresia Research and Education)
“We are deeply grateful to the patients and families who participated in EXPAND and helped move this research forward.”
Mirum Pharmaceuticals Announces Primary Endpoint Met in Phase 3 EXPAND Study of LIVMARLI(®) (maralixibat) in Ultra-Rare Cholestatic Liver Diseases
- Maralixibat demonstrated a statistically significant reduction in cholestatic pruritus compared with placebo (p=0.0004), and a 69% reduction in serum bile acids versus placebo (p<0.0001)
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