If you would like more information about our PFIC Phase 3 clinical trial called MARCH-PFIC, please visit www.pfictrial.com
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.
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.
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.
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 https://clinicaltrials.gov/ct2/show/NCT04530994.
No, the program is open to any patient > 12 months of age with a confirmed diagnosis of cholestatic pruritus associated with ALGS.
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.
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.
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.
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.
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: https://pfictrial.com.
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.