If you would like more information about our global 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 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.
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.
In the United States, 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.
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.
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 and abdominal pain, 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 in your region, Mirum will provide support in transitioning patients from the EAP to prescription use.
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.
The EAP is opening to eligible patients in Australia, Austria, Belgium, Canada, Denmark, France, Germany, Greece, Italy, Netherlands, Poland, Spain, Sweden, the United States, United Arab Emirates, and the United Kingdom.
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.
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