A New Drug Application (NDA) has been submitted by Eisai Medical Research, Inc., to the U.S. Food & Drug Administration (FDA) for the antiepileptic drug (AED) rufinamide. Eisai acquired exclusive North American and European manufacturing and marketing rights to rufinamide from Novartis Pharma, AG, in 2004.

Eisai is seeking approval for two indications for rufinamide, as adjunctive therapy for Lennox-Gastaut syndrome (LGS) in children ages 4 and over and as adjunctive therapy for partial-onset seizures with and without secondary generalization in adults and adolescents (12 years of age and over).

Rufinamide is a triazole derivative, a compound structurally distinct from currently approved AEDs. In a multicenter, double-blind, placebo-controlled clinical trial, rufinamide was shown to have clinical benefit in the treatment of seizures associated with LGS and was designated as an orphan drug for this indication by the FDA in October 2004. Lennox-Gastaut syndrome is a severe form of epilepsy. Seizures usually begin before 4 years of age, and children with LGS often suffer from several types of seizures. An estimated 1400 to 4500 new cases of LGS are diagnosed each year in the U.S., and complete recovery, including freedom from seizures and normal development, is very unusual. There is no known cure for the disorder.

Based on key findings of 2 double-blind, placebo-controlled trials, rufinamide will also be filed with the FDA as adjunctive therapy in adults and adolescents, 12 years of age and over, with partial-onset seizures with and without secondary generalization. Partial seizures affect only one area of the brain and are the most common type of seizure experienced by people with epilepsy.

In double-blind studies, the most commonly observed adverse experiences seen in association with rufinamide and at a higher frequency (greater than or equal to 10%) than in placebo-treated patients were headache, dizziness, fatigue, somnolence, and nausea.

How does the NDA Process Work?

The FDA defines the NDA application as "the vehicle through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing in the U.S. The data gathered during the animal studies and human clinical trials of an Investigational New Drug (IND) become part of the NDA.