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.