Mirum Pharmaceuticals sells a rare-disease medicine that faces growing pushback from insurers and governments, and could see tougher competition as protections around its key product fade. At the same time, faster international rollout, more people getting diagnosed, and progress in new treatments could keep growth going longer than skeptics expect.Read more
Mirum’s rare-disease medicines could reach far more people than most expect as better genetic testing and wider doctor awareness uncover patients who were previously missed. But the business leans heavily on a small set of treatments, so setbacks in approvals, pricing pushback, or new competition could hit results hard.Read more
Mirum Pharmaceuticals is growing as more patients get diagnosed and as its rare-disease medicine reaches new countries with insurance support. The next wave of clinical trial results could open new uses and new medicines, but the business also leans heavily on one product and faces pricing, approval, and competition risks.Read more
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Mirum Pharmaceuticals, Inc., a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as 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 and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases; Brelovitug, for the treatment of chronic HDV infection, which is in Phase 2 clinical trial; and MRM-3379, that is in Phase 2 clinical study for the treatment of FXS. The company has a collaboration agreement with the Shire International GmbH; License Agreement with Pfizer Inc.; Sanofi-Aventis Deutschland GmbH; Novartis Pharma AG; Lonza Ltd.; Enthorin Therapeutics, LLC and Dart Neuroscience LLC; Asset Purchase Agreement with Asklepion Pharmaceuticals, LLC; License and Manufacturing Agreement with LGM Pharma; and Asset Purchase Agreement with Travere Therapeutics, Inc. The company was incorporated in 2018 and is headquartered in Foster City, California.
