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

 

Now Opening in Select Regions Worldwide.

 

See Program Overview, Eligibility and Frequently Asked Questions Below

 

Physicians in the United States or Canada who are interested in the EAP should contact MirumALGS@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.

 

If you or a loved one have ALGS and reside in one of the above countries, please ask your physician to register for the program.

 

If you would like more information about our global PFIC Phase 3 clinical trial called MARCH-PFIC, please visit www.pfictrial.com

Program Overview:

Mirum’s investigational product, maralixibat, is being offered in select regions to eligible patients with cholestatic pruritus (itch) associated with ALGS.

The EAP is open for physician registration.

 

Physicians in the United States and Canada can email MirumALGS@clinigengroup.com to register for the EAP.

 

Physicians in Australia, Austria, Belgium, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, and the United Kingdom can email MedicineAccess@clinigengroup.com to register for the EAP.

Eligibility:

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 https://clinicaltrials.gov/ct2/show/NCT04530994.

Frequently Asked Questions – Patient and Family:

What is an EAP?

EAP stands for Expanded Access Program. These programs, conducted in compliance with applicable health authority 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 in select countries, 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?

In the United States, Belgium, Italy and Germany, the EAP program will be conducted under a protocol reviewed by the country regulatory authority. 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?

In countries where the EAP is available, 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 https://clinicaltrials.gov/ct2/show/NCT04530994.

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 and abdominal pain, 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 in your region, Mirum will provide support in transitioning patients from the EAP to prescription use.

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 conducting the EAP?

The EAP is opening to eligible patients in Australia, Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, the United States, and the United Kingdom.

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 global 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: https://pfictrial.com.

Where can I find more information?

More information can be found at the following website:

mirumpharma.com, or by emailing medinfo@mirumpharma.com.

If you are interested in being on the Mirum ALGS contact list for general informational updates, please enter your contact information below.

* Indicates required field.
  • Contact Name: