Expanded Access Program

Marinus Pharmaceuticals supports Expanded Access Programs (EAPs) as an important way of allowing patients access to investigational medicines prior to FDA approval. 

Expanded Access Treatment Protocol For Ganaxolone

Expanded access, sometimes called “compassionate use,” is the use of an investigational medicine (i.e., one that has not been approved by the Food and Drug Administration (FDA)) by a patient who can not be enrolled in a clinical trial.

Why does Marinus have an EAP?

An EAP is intended to allow patient access to an investigational medicine for a serious or immediately life-threatening disease or condition where no comparable or satisfactory alternative therapy exists and when the potential benefit of the medicine justifies the potential risks and those risks are not unreasonable in the context of the disease or condition being treated. One type of EAP is an Expanded Access Treatment Protocol, which provides access to an investigational medicine to a larger population.

It is important to remember that investigational drugs have not yet received regulatory approval; therefore, their potential risks and benefits are not yet established. Doctors and patients should consider all possible benefits and risks when seeking expanded access to an investigational drug.

Click here to watch our Expanded Access Program FAQ video

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How to access the program

Physicians may submit requests to participate in the Expanded Access Treatment Protocol for ganaxolone on behalf of patients in their care.

Parents and other caregivers may submit questions about the Expanded Access Treatment Protocol for ganaxolone.

Click here to visit our web portal

After a physician requests a patient’s participation, the process for review of the request for appropriateness and initiation into the Expanded Access Treatment Protocol for Ganaxolone may take up to 4 weeks. For new patients enrolled in the Expanded Access Treatment Protocol for Ganaxolone, the protocol requires the capture of baseline data prior to treatment which may take as long as 28 days.

Expanded Access Program FAQ