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.
If you or a loved one have PFIC, please ask your physician to register for the program.
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.
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?
Is the EAP a research study?
Are there any eligibility restrictions for the program?
Is this program only for pediatric patients?
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.