Mirum Pharmaceuticals, Inc. (08D)

Overview

Mirum Pharmaceuticals, Inc. is a pioneering biopharmaceutical company with a sharp focus on the discovery, development, and commercialization of innovative therapies designed to tackle debilitating rare and orphan diseases. Established in 2018 and based in Foster City, California, Mirum Pharmaceuticals has swiftly moved to the forefront of its field, aiming to deliver significant medical advancements for conditions with limited treatment options. The company's strategic approach combines breakthrough science with an unwavering commitment to improving patient outcomes, positioning it as a leader in rare disease therapy development.

Products and Services

• LIVMARLI (maralixibat)

  This flagship product stands out as Mirum Pharmaceuticals' premier offering. LIVMARLI is an orally administered, minimally absorbed ileal bile acid transporter (IBAT) inhibitor. It has gained approval for the treatment of cholestatic pruritus in patients suffering from Alagille syndrome both in the United States and on an international scale. LIVMARLI's innovative mechanism offers new hope for patients grappling with this challenging and rare condition, illustrating the company's dedication to filling critical treatment gaps in rare diseases.

• Cholbam

  A cholic acid capsule, Cholbam is another critical component of Mirum Pharmaceuticals' product lineup. The U.S. Food and Drug Administration (FDA) has approved it for use in both pediatric and adult patients who are dealing with bile acid synthesis disorders resulting from single enzyme defects. Furthermore, Cholbam is sanctioned as an adjunctive treatment for various peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome. This approval underscores the company's commitment to addressing unmet needs across a spectrum of rare bile acid disorders.

• Chenodal

  Approved for the treatment of radiolucent stones in the gallbladder, Chenodal is a key drug in Mirum Pharmaceuticals' suite of products. Beyond its established use, Chenodal is currently under Phase 3 development for addressing cerebrotendinous xanthomatosis, showcasing the company's ongoing efforts to expand its impact across additional rare diseases through rigorous clinical development.

• Volixibat

  Mirum Pharmaceuticals is also advancing Volixibat, a promising therapeutic under investigation. Like LIVMARLI, Volixibat is designed to inhibit the IBAT through an oral, minimally absorbed formulation. Currently in a Phase 2b clinical trial, Volixibat targets the treatment of adult patients with cholestatic liver diseases, signifying the company's continuous pursuit of innovative solutions for complex liver conditions.