Ganaxolone is a steroid

Steroid

A steroid is a type of organic compound that contains a characteristic arrangement of four cycloalkane rings that are joined to each other. Examples of steroids include the dietary fat cholesterol, the sex hormones estradiol and testosterone, and the anti-inflammatory drug dexamethasone.The core...

 drug, with the molecular formula C22H36O2, related to allopregnanolone

Allopregnanolone

Allopregnanolone is a prototypic neurosteroid present in the blood and also the brain. It is a metabolite of progesterone and potent modulator of GABAA receptors...

 that has sedative

Sedative

A sedative or tranquilizer is a substance that induces sedation by reducing irritability or excitement....

, anxiolytic

Anxiolytic

An anxiolytic is a drug used for the treatment of anxiety, and its related psychological and physical symptoms...

, and anticonvulsant

Anticonvulsant

The anticonvulsants are a diverse group of pharmaceuticals used in the treatment of epileptic seizures. Anticonvulsants are also increasingly being used in the treatment of bipolar disorder, since many seem to act as mood stabilizers, and in the treatment of neuropathic pain. The goal of an...

 effects. It is a potent and selective positive allosteric modulator of GABA_A receptors

Receptor (biochemistry)

In biochemistry, a receptor is a molecule found on the surface of a cell, which receives specific chemical signals from neighbouring cells or the wider environment within an organism...

. Ganaxolone protects against seizures in diverse animal models, including the 6 Hz and amygdala kindling models. Unlike for benzodiazepines, there is no tolerance to the anticonvulsant effects of ganaxolone

Ganaxolone is being investigated for potential medical use in the treatment of epilepsy

Epilepsy

Epilepsy is a common chronic neurological disorder characterized by seizures. These seizures are transient signs and/or symptoms of abnormal, excessive or hypersynchronous neuronal activity in the brain.About 50 million people worldwide have epilepsy, and nearly two out of every three new cases...

. It is well tolerated in human trials (with exposure of >900 patients), with the main side-effects being sedation, dizziness

Dizziness

Dizziness refers to an impairment in spatial perception and stability. The term is somewhat imprecise. It can be used to mean vertigo, presyncope, disequilibrium, or a non-specific feeling such as giddiness or foolishness....

, and headache

Headache

A headache or cephalalgia is pain anywhere in the region of the head or neck. It can be a symptom of a number of different conditions of the head and neck. The brain tissue itself is not sensitive to pain because it lacks pain receptors. Rather, the pain is caused by disturbance of the...

. Trials in adults with partial seizures and in infantile spasms have recently been completed.