Maralixibat Expanded Access Program for Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)

 

Now Opening in Select Regions Worldwide.

 

SEE PROGRAM OVERVIEW, ELIGIBILITY AND FREQUENTLY ASKED QUESTIONS BELOW

Physicians in Canada and the US who are interested in the EAP should contact MirumPFIC@clinigengroup.com to register.

Physicians outside of Canada and the US who are interested in the EAP should contact MedicineAccess@clinigengroup.com to register.

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

PROGRAM OVERVIEW:

Mirum’s product, maralixibat, which is currently being clinically studied in patients with PFIC, is also being made available to eligible patients in select regions.

The EAP is open for physician registration.

Physicians in Canada and the US can email MirumPFIC@clinigengroup.com to register for the EAP.

Physicians outside of Canada and the US can email MedicineAccess@clinigengroup.com to register for the EAP.

ELIGIBILITY:

Mirum’s EAP is open to patients with PFIC who are >3 months of age and >5kg who have no other therapeutic options and do not have access to an ongoing PFIC clinical trial.

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 Progressive Familial Intrahepatic Cholestasis (PFIC) 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 PFIC and be greater than 3 months in age and greater than 5kg. Maralixibat will be provided at no cost until it becomes available for prescription use.
Is the EAP a research study?
In 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 PFIC patients are managed in clinical practice. Outside of Germany and Italy, the access to maralixibat is managed via a Named Patient Program.
Are there any eligibility restrictions for the program?
In countries where the EAP is available, patients >3 months of age and >5kg with a confirmed diagnosis of PFIC who do not have access to an ongoing PFIC clinical trial will be eligible for this EAP. Patients must be evaluated by their physician for eligibility.
Is this program only for pediatric patients?
No, the program is open to any patient >3 months of age and >5kg with a confirmed diagnosis of PFIC.
What is the most important information I should know about maralixibat?

Maralixibat has been studied in over 1,600 patients and over 150 pediatric patients. Maralixibat was generally well-tolerated in clinical studies. LIVMARLI (maralixibat) has been approved by the FDA for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 1 year of age and older.

LIVMARLI can cause serious side effects, including:

  • Changes in liver tests: Changes in certain liver tests are common in patients with Alagille syndrome but may worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your health care provider should do blood tests before starting and during treatment to check your liver function.
  • Stomach and intestinal (gastrointestinal) problems: LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment.
  • Fat Soluble Vitamin Deficiency: This vitamin deficiency is common in patients with Alagille syndrome but may worsen during treatment.
Will the medicine be covered by my insurance company?
While participating in the EAP for PFIC, 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 150 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 PFIC EAP is opening to eligible patients in US, Canada, many countries in Europe, Latin America and the United Arab Emirates.
Where can I find more information?

More information can be found at the following website: mirumpharma.com, or by emailing medinfo@mirumpharma.com.