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 Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC), 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 clinicaltrials@mirumpharma.com.

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Hear From Patients, Families & Clinicians

At Mirum, we work closely with patient communities in every part of our business, from disease awareness and education to clinical trial design. Hear from patients, families, and clinicians as they share their perspectives on the diseases and the need for a transformative medicine.

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. For more information about how to enroll in our clinical trials, please contact us at clinicaltrials@mirumpharma.com.

Access to Mirum’s Therapies

Expanded Access Policy Statement

Clinical development is a critical part of evaluating experimental therapies as potential impactful medicines for people with rare cholestatic liver diseases. We are committed to rigorous testing of experimental treatments with the goal of securing regulatory approval and enabling the medicine to be available to as many patients as quickly as possible.

Expanded Access Program for Alagille syndrome

Mirum’s Expanded Access Program (EAP) for Alagille syndrome (ALGS) is now open in the United States and Canada, offering access to maralixibat for the treatment of cholestatic pruritus in eligible patients with ALGS who do not have access to ongoing clinical trials.

Requests to participate in this program must be made by a licensed physician. Physicians and patients can learn more about the maralixibat EAP for ALGS by visiting the program website at www.ALGSEAP.com or clinicaltrials.gov/ct2/show/NCT04530994 or contact MirumALGS@clinigengroup.com.

Unfortunately, we are not able to offer expanded access to our investigational treatments in diseases other than ALGS, or in regions outside of the US and Canada, at this time. We will periodically reevaluate this policy as new data become available.

To learn more about Mirum’s open clinical trials, please visit Clinical Trials at Mirum, pfictrial.com or clinicaltrials.gov.

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