Patients & Families

Mirum is focused on developing well-tolerated and effective treatments for children and adults with rare cholestatic liver diseases. What we want, more than anything, is to enable patients and their families to live healthier, longer lives 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), and biliary atresia, for which there are no approved therapies. We are also evaluating volixibat for the treatment of primary sclerosing cholangitis (PSC), intrahepatic cholestasis of pregnancy (ICP), and primary biliary cholangitis (PBC).

Genetic Testing

Mirum Pharmaceuticals is working alongside Travere Therapeutics (formerly Retrophin) to provide, to qualified patients at no charge, the EGL Genetic Cholestasis Panel. This panel may assist in diagnosing a child with a rare genetic liver disease. 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 work on behalf of patients and we partner with them. Through clinical trials for our investigational medicines, patients and their families play an integral role in helping us evaluate and obtain the evidence we need for regulatory approval in the U.S. and beyond.

Studies currently enrolling include:

MARALIXIBAT

Progressive Familial Intrahepatic Cholestasis: Phase 3 MARCH study

Evaluating the safety and efficacy of maralixibat in pediatric patients with progressive familial intrahepatic cholestasis.

Clinicaltrials.gov identifier: NCT03905330

Biliary Atresia: Phase 2b EMBARK study

Evaluating maralixibat in pediatric patients with biliary atresia post-Kasai procedure.

Clinicaltrials.gov identifier: NCT04524390

Cholestatic Liver Diseases: Phase 2 RISE study

Evaluating the safety and tolerability of maralixibat in infants 12 months or younger with PFIC and ALGS

Clinicaltrials.gov identifier: NCT04729751

Alagille syndrome: Expanded Access Program

The expanded access program is to provide access to maralixibat for eligible patients with cholestatic pruritus associated with Alagille syndrome.

For more information about the study including how to enroll, please visit the EAP website at algseap.com.

Clinicaltrials.gov identifier: NCT04530994

VOLIXIBAT

Primary Sclerosing Cholangitis: Phase 2b VISTAS study

Evaluating the safety and efficacy of volixibat in patients with pruritus (itch) caused by primary sclerosing cholangitis.

Clinicaltrials.gov identifier: NCT04663308

Intrahepatic Cholestasis of Pregnancy: Phase 2a/2b OHANA study

Evaluating volixibat in pregnant women with elevated serum bile acids associated with intrahepatic cholestasis of pregnancy.

Clinicaltrials.gov identifier: NCT04718961

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 open in Australia, Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, the United States, and the United Kingdom, 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 in the United States or Canada who are interested in the EAP should contact Mirum ALGS@clinigengroup.com to register.

Physicians in Australia, Austria, Belgium, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, and the United Kingdom who are interested in the EAP should contact MedicineAccess@clinigengroup.com to register.

Physicians and patients can learn more about the maralixibat EAP for ALGS by visiting the program website at ALGSEAP.com or Clinical Trials.gov.

At this time, we are not able to offer expanded access to our investigational treatments in diseases other than ALGS, or regions other than those listed above.

To learn more about Mirum’s open clinical trials, please visit the Mirum website, pfictrial.com or clinicaltrials.gov.