Mirum Pharmaceuticals develops targeted therapies for rare diseases, focusing on transforming scientific discoveries into effective treatments. The company addresses the unmet medical needs of patients and families affected by these conditions, aiming to improve their quality of life through innovative biopharmaceutical solutions.
Funding
$275M raised to dateRaised to date based on public sources. This may differ from the amount the company actually raised and is based only on what is publicly available on the internet.
Founders
Product
Problem
Many rare diseases, particularly those affecting the liver and bile acid metabolism, lack effective treatments, leaving patients with limited options and significant unmet medical needs. Diagnosing these conditions can also be challenging, leading to delays in appropriate care and management.
Solution
Mirum Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing therapies for rare liver diseases. Their approach involves identifying and developing innovative treatments that target the underlying mechanisms of these diseases. Mirum's portfolio includes therapies aimed at improving outcomes for patients with cholestatic liver diseases and inborn errors of bile acid metabolism. They also offer diagnostic testing programs to facilitate earlier and more accurate diagnoses.
Target Audience
The primary target audience includes patients with rare liver diseases, their families, and healthcare professionals specializing in hepatology and gastroenterology.
Features
- LIVMARLI: An approved treatment for cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).
- Cholbam: A treatment for bile acid synthesis disorders due to single enzyme defects and peroxisomal disorders.
- CTEXLI™: Investigational therapy for rare liver diseases.
- Diagnostic testing programs: Facilitate the identification of patients with specific rare liver diseases.
- Focus on cholestasis and inborn errors of bile acid metabolism.