Innovating for Epilepsy and Rare Disease Patients

Ganaxolone – A Novel GABAA Receptor Modulator

Our goal is to maximize the potential of ganaxolone to address areas of unmet medical need where its mechanism can have the greatest impact, including a range of seizure disorders. Ganaxolone possesses enhanced activity at receptors for the neurotransmitter GABA (gamma-aminobutyric acid), the major regulator of excessive activity of neurons in the brain. It may have the potential to modulate the abnormalities that underlie some of the most severe seizure disorders.


Marinus is developing ganaxolone formulations, both intravenous (IV) and oral, to maximize its therapeutic potential in both acute care and outpatient settings for adult and pediatric patients.
We are committed to continuing to optimize the ganaxolone formulations to keep pace with innovation and are currently developing a second generation oral formulation and prodrug.

Ganaxolone has been studied in more than 1,900 patients, both children and adults

How Ganaxolone Works

Ganaxolone is a GABAA receptor modulator that acts by regulating brain activity. This process can include inhibiting the abnormal electrical discharges that cause seizures and status epilepticus or restoring balance in disrupted neuronal activity in other CNS disorders.

Because ganaxolone modulates both synaptic and extrasynaptic GABAA receptors and is unique relative to other approved antiepileptic drugs, we believe it has the potential to treat seizures resistant to other available medications. These binding sites, including the extrasynaptic GABAA receptor, may also be important in treating status epilepticus and other brain disorders.