Key Takeaways Expanded patient identification and accelerated adoption of therapies are driving growth beyond market expectations, with international markets providing significant additional revenue potential. Advances in diagnostics and a strong financial position enable Mirum to extend treatment durations, acquire new assets, and maintain robust profitability and pricing power.Read more
Key Takeaways Broader patient identification, international expansion, and supportive reimbursement environments are driving sustained revenue growth and improving margins for Mirum's portfolio. Advancing late-stage pipeline programs and leveraging precision medicine support future product expansion, revenue diversification, and long-term earnings acceleration.Read more
Key Takeaways Intensifying cost controls and payer scrutiny threaten long-term revenue, especially as rare disease therapy reimbursement becomes increasingly difficult in US and international markets. Competitive pressures from generics, shifting treatment paradigms, and high regulatory risk will limit market share, profitability, and future revenue growth.Read more
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 Phase 3 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. The company was incorporated in 2018 and is headquartered in Foster City, California.
