Status Epilepticus (SE) 

Our mission: Provide new treatment options for patients with SE.

At Marinus, we are developing ganaxolone IV for the hospital setting, offering a new mechanism of action for SE patients who continue to experience seizures despite treatment.

What is Status Epilepticus?

Status epilepticus (SE) is a life-threatening occurrence of continued or intermittent seizures lasting more than five minutes in duration without recovery of consciousness. SE is the second most common neurological emergency and is associated with significant mortality and morbidity. New treatment options that rapidly stop the seizures and prevent escalation of treatment are needed to improve outcomes.

Marinus CEO Dr. Scott Braunstein provides an overview of status epilepticus


The science behind treating SE

Seizures persist when neuronal networks are over-excited. GABAergic neurons aim to inhibit, or calm, these networks during ongoing or impending seizures. Receptors at the end of these neurons (synapse) decrease as a result of persistent seizures, including status epilepticus. As a result, drugs that act solely on these receptors (synaptic GABAA receptors), such as benzodiazepines, become ineffective over time.

However, there are GABAA receptors that reside just outside of the synapse (extrasynaptic) and mediate tonic inhibition. Unlike synaptic GABAA receptors, extrasynaptic GABAA receptors remain available for drug targeting during status epilepticus.

Ganaxolone’s differentiated mechanism of action works by increasing GABAergic inhibition through both types of GABAA receptors making it unique to other antiseizure medications used in the treatment of status epilepticus.

Rapid onset of action

Another goal for new SE treatment options is a rapid onset of action. IV ganaxolone, due to its chemical properties, has been shown in pre-clinical and clinical studies to rapidly enter and act on the networks within the brain within minutes.

The target formulation is intravenous. IV ganaxolone allows for rapid administration in emergency situations, can quickly act on seizures, and allows for well-controlled dosing. IV ganaxolone has received orphan drug designation from the U.S. FDA for the treatment of SE.

Emergency Room, Compressed

It has been estimated that up to 150,000 cases of SE occur annually in the U.S.

The clinical development of ganaxolone in SE

An open-label Phase 2 study in 17 patients provided preliminary evidence that IV ganaxolone may be effective at rapidly stopping status epilepticus and preventing escalation to third-line IV anesthetics in patients with refractory SE.

A randomized, placebo-controlled Phase 3 study, the RAISE study, in refractory SE is ongoing.  The RAISE study aims to assess the rapid onset of action and durability of effect as well as safety.

Status Epilepticus Er Team

Learn more about status epilepticus

Additional educational and support resources:

Centers for Disease Control

Epilepsy Foundation

National Institute for Neurological Disorders and Stroke

International League Against Epilepsy

Links to third party sites are provided for convenience purposes only. The information contained on these sites is not information provided, controlled or monitored by Marinus Pharmaceuticals in any way. Marinus Pharmaceuticals is not responsible in any way for the accuracy, completeness, or fitness for any particular purpose of any content appearing on such sites.