Maralixibat is an orally-administered liquid. Maralixibat is an investigational medicine being evaluated as a treatment for children with rare cholestatic liver diseases, including PFIC.
Maralixibat works by blocking an important bile acid transport protein on the surface of the small bowel, thereby reducing systemic levels of bile acids.
Encouraged by promising Phase 2 clinical data, Mirum Pharmaceuticals is now advancing maralixibat into Phase 3 clinical studies. To date, more than 119 children and 69 adults have received maralixibat as a treatment for their cholestatic liver diseases, many of them for more than 4 years. Maralixibat has also been tested in > 1400 subjects in other studies.
Results from the Phase 2 INDIGO study supported maralixibat’s Breakthrough Therapy Designation for the treatment of PFIC2 from the US FDA. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs by the FDA that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.