Mirum Pharmaceuticals, Inc.

We are a biopharmaceutical company focused on treating rare diseases. We have three approved medicines: LIVMARLI® (maralixibat), CHOLBAM® (cholic acid) capsules, and CTEXLI® (chenodiol) tablets. Livmarli is an orally administered, minimally-absorbed ileal bile acid transporter (IBAT) inhibitor. It is approved for cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S., the European Union (EU), and various other countries. Livmarli is also approved for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) in the U.S., Canada, and Japan, and for the treatment of PFIC in the EU. We market Livmarli in the U.S., Canada, and certain European countries through our commercial team and have license and distribution agreements for other countries. We are also conducting the EXPAND study to add indications for Livmarli for cholestatic pruritus due to other rare conditions. In August 2023, we acquired assets from Travere Therapeutics, Inc. related to the development, manufacture, and commercialization of chenodiol and Cholbam (also known as Kolbam). The U.S. Food and Drug Administration (FDA) approved Cholbam in March 2015 as the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder (PBD-ZSD). Chenodiol is the standard of care for cerebrotendinous xanthomatosis (CTX) in the U.S. and was commercialized as Chenodal. We submitted a New Drug Application (NDA) for chenodiol for CTX in 2024 and received FDA approval for the treatment of adults with CTX in February 2025.