Maralixibat Expanded Access Program for Patients with Alagille Syndrome (ALGS)


The program is available in the U.S. and Canada.


See Program Overview, Eligibility and Frequently Asked Questions Below


Physicians interested in the EAP should contact to register. You must be a physician in the US or Canada to register.


If you or a loved one have ALGS, please ask your physician contact to register for the program.


If you are outside the US or Canada, please check this website for updates to the program.
You may also complete the form below.


If you would like more information about our PFIC Phase 3 clinical trial called MARCH-PFIC, please visit

Program Overview:

Mirum’s investigational product called maralixibat is being offered in the United States and Canada to eligible patients with ALGS with cholestatic pruritus (itch).

The EAP is open for physician registration. Physicians can email to register for the EAP.


Mirum’s EAP is open to patients with ALGS who are >12 months old with clinically significant (e.g. moderate to severe) cholestatic pruritus and who do not have access to an ongoing ALGS clinical trial. Additional details can be found at

Frequently Asked Questions – Patient and Family:

What is an EAP?

EAP stands for Expanded Access Program.  These programs, conducted in compliance with FDA and Health Canada guidance, are designed to facilitate patient access to products (drugs, biologics, or medical devices) for those with life-threatening diseases where no approved treatment options currently exist.

What is the Mirum Alagille syndrome (ALGS) EAP?

For eligible patients, Mirum’s EAP provides access to maralixibat prior to regulatory approval. Eligible patients must have a confirmed diagnosis of cholestatic pruritus associated with ALGS and be greater than 12 months in age. Maralixibat will be provided at no cost until it becomes available for prescription use.

Is the EAP a research study?

The EAP program will be conducted under a protocol.  Therefore, informed consent is required. Patient data will be collected such as: height, weight, and various lab values.  This data will be captured as a part of standard clinical practice. The collection of this data will allow Mirum to better understand how ALGS patients are managed in clinical practice.

Are there any eligibility restrictions for the program?

The program will initially open in the United States and Canada as we explore expansion to other countries. Patients >12 months of age with a confirmed diagnosis of cholestatic pruritus associated with ALGS who do not have access to an ongoing ALGS clinical trial will be eligible for this EAP. Patients must be evaluated by their physician for eligibility. Additional details can be found at

Is this program only for pediatric patients?

No, the program is open to any patient > 12 months of age with a confirmed diagnosis of cholestatic pruritus associated with ALGS.

What is the most important information I should know about maralixibat?

Maralixibat has been studied in over 1,600 patients and over 120 pediatric patients.  Maralixibat was generally well-tolerated in clinical studies. The main side effects experienced were diarrhea, abdominal pain and nausea, of mild to moderate severity and usually temporary.

Will the medicine be covered by my insurance company?

While participating in the EAP for ALGS, maralixibat will be provided at no cost to patients.  If maralixibat is approved for prescription use, at that time, Mirum will have an external team working with patients to assist with insurance options.

How long can I/my child take maralixibat?

Maralixibat has been studied in more than 1,600 patients –including more than 120 children.  Some patients have received maralixibat for longer than 5 years.  How long you or your child continues to be treated with maralixibat will depend on tolerability and therapeutic goals, as determined by your treating physician.

In what countries are you planning on conducting the EAP?

As currently planned, the EAP will be available to eligible patients in the United States and Canada, beginning September 2020. The team at Mirum continues to explore opening the EAP in other parts of the world.

My child has Progressive Familial Intrahepatic Cholestasis (PFIC). What access to maralixibat is available?

For patients with PFIC, access to maralixibat is possible through our MARCH Phase 3 study, which is currently open to enrollment. Mirum continually evaluates the need for expanded access to maralixibat as studies reach enrollment milestones. More information can be found at:

When will maralixibat become available for doctors to prescribe?

Mirum is preparing to file a New Drug Application (NDA) with the FDA for ALGS patients with cholestatic pruritus. The FDA will likely complete their review in the second half of 2021.

Where can I find more information?

More information can be found at the following websites: , Or by emailing

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