Ganaxolone Indications

Infantile Spasms

Infantile spasms, also known as West's Syndrome, is a catastrophic pediatric epilepsy disorder that affects as many as 2000 new children in the U.S. each year. The onset is predominantly in the first year of life, typically between 3 to 6 months. Infantile spasms seizures often consist of a sudden bending forward and stiffening of the body, arms, and legs ('jacknife'), sometimes with arching of the torso. Individual spasms typically last for 1 to 5 seconds and occur in clusters, ranging from 2 to 100 spasms at a time. Infants may have dozens of clusters and several hundred spasms per day. Infantile spasms may stop by age 5, but are often replaced by other seizure types, such as the Lennox-Gastaut syndrome. Infantile spasms is almost invariably associated with mental retardation (95%) and in some cases mortality (5-20%).

Adult Partial Complex Seizures

Approximately 2 million people in the U.S. have epilepsy, and complex partial seizures occur in about 35% of persons with epilepsy Complex partial seizures result in impaired consciousness, with symptoms like decreased responsiveness and awareness of self and surroundings. During a complex partial seizure, the patient may not communicate, respond to commands, or remember events that occurred, although some patients may make simple responses, follow simple commands, or continue to perform simple or, less commonly, complex motor behaviors such as operating a car. Complex partial seizures typically arise from the temporal lobe of the brain but may arise from any cortical region. As many as 30% of epilepsy patients may not be well controlled on treatment with a single or multiple existing antiepileptic drugs (French et al., 2004). The approach of adding on an experimental antiepileptic drug to an existing non-optimal therapeutic regimen has proven to be a useful technique for demonstrating efficacy of novel compounds. The planned study is designed to evaluate the safety, tolerability, and efficacy, of ganaxolone when used as an add-on therapy in adult patients with partial seizures who are uncontrolled on their present AED regimen.

French JA, Kanner AM, Bautista J, Abou-Khalil B, Browne T, Harden CL, Theodore WH, Bazil C, Stern J, Schachter SC, Bergen D, Hirtz D, Montouris GD, Nespeca M, Gidal B, Marks WJ, Jr., Turk WR, Fischer JH, Bourgeois B, Wilner A, Faught RE, Jr., Sachdeo RC, Beydoun A, Glauser TA (2004) Efficacy and tolerability of the new antiepileptic drugs II: treatment of refractory epilepsy: report of the Therapeutics and Technology Assessment Subcommittee and Quality Standards Subcommittee of the American Academy of Neurology and the American Epilepsy Society. Neurology 62:1261-1273.