SK Biopharmaceuticals receives FDA Orphan Drug status for carisbamate

SK Biopharmaceuticals, a South Korean pharmaceutical company, announced today that it was granted special status by the U.S. Food and Drug Administration (FDA) for carisbamate for the management of patients with infantile spasms (IS), a form of epilepsy associated with increased risk of death or mental retardation.    

Dr. Christopher Gallen, CEO of SK Biopharmaceuticals, said, "Carisbamate is an important new anti-seizure therapy. Experimental evidence demonstrates its utility in a model of IS. The designation of carisbamate as an Orphan Drug by the FDA facilitates our progression of this vital medication to yet another area of great unmet medical need."

The FDA grants Orphan Drug designation to products that are intended to treat rare or orphan diseases or conditions such as infantile spasms. For IS patients, as with patients fighting other rare diseases, new treatments are few and far between, making this designation by the FDA of significant importance.

"IS, or West Syndrome, is a devastating and difficult to treat form of childhood epilepsy that often leads to some form of mental retardation and other behavioral problems over time," said Dr. Jeongwoo Cho, Vice President of SK Biopharmaceuticals' Drug Development Business. "Carisbamate is being developed to provide physicians with an additional treatment option for patients with IS for whom there are very few safe and effective treatments."

Carisbamate has been designated as an orphan drug by the FDA under the auspices of the Orphan Drug Act. The Act, which will mark its 30^th anniversary on January 4, 2013, provides incentives to sponsors that develop therapies intended to treat orphan conditions, which are classified as any condition or disease that affects less than 200,000 patients per year in the U.S. It is estimated that there are more than 6,000 rare diseases affecting 25 million Americans.

Carisbamate has previously been shown to have efficacy in clinical trials for adult forms of epilepsy, such as partial onset seizures.