Maralixibat

Unassigned

New Medicines

Progressive familial intrahepatic cholestasis (PFIC) in children

Information

New molecular entity
Mirum
Mirum

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Potent and selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT)
Estimated prevalence of PFIC varies between 1 in 50,000 and 1 in 100,000
Progressive familial intrahepatic cholestasis (PFIC) in children
Oral

Evidence based evaluations