Patients & Families

Mirum is focused on developing a safe and effective treatment for children with rare cholestatic liver diseases. What we want, more than anything, is to enable patients and their families to live a healthy and happy life, liberated from around-the-clock disruptions of disease.

We are currently advancing maralixibat for the treatment of progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS), for which there are no approved therapeutics.

Genetic Testing

Mirum Pharmaceuticals is partnering with Retrophin, Inc., to provide, to qualified patients at no charge, the EGL Genetic Cholestasis Panel. This panel may assist in diagnosing a child who has one of these cholestatic liver diseases. For more information about how to access this program, please contact


Clinical Trials

We don’t just work on behalf of patients, we work with them. Through clinical trials for our investigational medicine, maralixibat, patients and their families play an integral role in helping us evaluate the drug and obtain the evidence we need for regulatory approval in the U.S. and beyond.

We initiated the Phase 3 MARCH study of maralixibat in children with PFIC in July 2019. We are in discussions with regulatory agencies with respect to the adequacy of our existing data to support regulatory submissions of maralixibat for the treatment of pruritus associated with ALGS in children. For more information about how to enroll in our clinical trials, please contact us at

Compassionate Use

Expanded Access (“Compassionate Use” or “Early Access”) Statement

Clinical development is a critical part of evaluating experimental drugs as potential impactful medicines for children with rare cholestatic liver diseases. Our ultimate goal is the rigorous testing of the experimental drugs with the aim of securing regulatory approval and enabling the medicine to be available to as many patients as possible as quickly as possible. With this in mind, Mirum does not offer our unapproved medicines via expanded access at this time.  Mirum believes that participation in one of our clinical trials is the most appropriate way to access our experimental drugs. Therefore, we encourage interested patients to consider being evaluated for entry into one of Mirum’s clinical trials. For additional information about this policy, please contact us at

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