Mirum Expanded Access Programs

Clinical development is a critical part of evaluating experimental therapies as potential impactful medicines for people with rare liver diseases. We are committed to rigorous testing of experimental treatments in order to secure regulatory approval and enable the medicine to be available to as many patients as quickly as possible.

EAP stands for Expanded Access Program. These programs, conducted in compliance with applicable health authority guidance, are designed to facilitate patient access to investigational products (drugs, biologics, or medical devices) for those with life-threatening diseases where no approved treatment options currently exist.

Maralixibat

Maralixibat is a minimally absorbed, orally administered liquid. It’s an investigational medicine being evaluated as a treatment for rare liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and biliary atresia (BA).

LIVMARLI® (maralixibat) has been approved by the U.S. FDA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older.

European Commission has granted marketing authorization for LIVMARLI® (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) two months of age and older. Maralixibat is available under an EAP for ALGS patients in a selection of countries and for PFIC in select regions worldwide.

Find more details on our current Maralixibat Expanded Access Programs.

Alagille Syndrome (ALGS)

Physicians who are interested in the EAP should contact:

Canada:

MirumALGS@clinigengroup.com

Rest of the world:

MedicineAccess@clinigengroup.com

Progressive Familial Intrahepatic Cholestasis (PFIC)

PFIC EAP HOME