Did you know that it is now possible to work with your local physician to access ganaxolone in the US? 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 cannot be enrolled in a clinical trial.
We asked Marinus to tell us more about their new Expanded Access Program (EAP) for ganaxolone. We think they answer many of the questions you might have in this video. Please visit the EAP portal to learn more about how your local care team can request access to this investigational drug for your loved one living with CDD.
Last year, Marinus reported positive topline data from the Phase 3 Marigold Study, the first double-blind placebo-controlled trial to provide evidence of efficacy in CDD and the first Phase 3 trial to examine three times a day dosing of ganaxolone in pediatric patients. You can read more at this link.