Mirum Pharmaceuticals, Inc. is a biopharmaceutical company focused on the development and commercialization of innovative therapies for rare and orphan diseases, particularly those affecting liver function and metabolic processes. The company’s mission is to bring transformative medicines to patient populations with significant unmet medical needs, leveraging deep expertise in rare disease science and close engagement with disease communities.

Mirum’s commercial portfolio includes LIVMARLI® (maralixibat)—an orally administered inhibitor of the ileal bile acid transporter approved to treat cholestatic pruritus in conditions such as Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC); CHOLBAM® (cholic acid) for bile acid synthesis disorders; and CTEXLI™ (chenodiol) for cerebrotendinous xanthomatosis (CTX) and related indications.

In addition to its marketed products, Mirum maintains a diverse clinical-stage pipeline targeting other rare diseases, including agents in late-stage development for primary sclerosing cholangitis (PSC), primary biliary cholangitis (PBC), and Fragile X syndrome. The company combines strategic scientific development with purposeful collaboration across the rare disease ecosystem to advance high-impact medicines from early research through regulatory approval and patient access.