Mirum Pharmaceuticals is advancing its late-stage pipeline with two investigational treatments aimed at debilitating liver diseases. Volixibat, an IBAT inhibitor, is currently being evaluated in two potentially registrational studies: the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and the Phase 2b VANTAGE study for primary biliary cholangitis. Notably, Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC.
In addition to Volixibat, chenodiol has been the focus of a Phase 3 clinical study, RESTORE, aimed at treating patients with CTX. The study reported positive topline results in 2023, leading Mirum to submit a new drug application to the FDA for the approval of chenodiol to treat CTX in the U.S.
Mirum Pharmaceuticals continues to explore innovative treatments for liver diseases, demonstrating a commitment to addressing unmet medical needs in this area.
