Mirum Pharmaceuticals
Mirum Pharmaceuticals specializes in developing therapies for rare liver diseases and offers three FDA-approved medications: LIVMARLI, Cholbam, and Chenodal, alongside conducting clinical trials for investigational treatments.
Services
Mirum Pharmaceuticals provides genetic testing for cholestasis at no cost to patients, aiding in the diagnosis of cholestatic liver diseases. The company also offers the Mirum Access Plus program, which assists patients and families with treatment access and financial support. Their services extend to global regulatory and clinical development efforts aimed at bringing life-saving therapies to patients across the world.
Approved Medications
Mirum Pharmaceuticals has three approved medications. LIVMARLI is approved for treating cholestatic pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestasis. Cholbam is used to treat bile acid synthesis disorders due to single enzyme deficiencies and as an adjunctive treatment for peroxisomal disorders. Chenodal has been determined by the FDA to be medically necessary for treating cerebrotendinous xanthomatosis.
Clinical Trials
Mirum Pharmaceuticals is actively conducting clinical trials for investigational treatments such as volixibat. These trials focus on conditions including primary sclerosing cholangitis and primary biliary cholangitis. The company's clinical trials aim to expand the range of available therapies for rare liver diseases.
Pipeline
Mirum Pharmaceuticals' pipeline includes treatments targeting rare liver diseases that affect both children and adults. The company's ongoing research and development efforts are designed to address unmet medical needs in this field, providing hope for patients with limited treatment options.