Insys Therapeutics Inc., a specialty pharmaceutical company that is developing and commercializing innovative drugs and novel drug delivery systems, announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its pharmaceutical cannabidiol (CBD) for the treatment of Dravet syndrome, a rare pediatric-onset epilepsy.
"There is presently no cure for this catastrophic form of epilepsy, and the significant, unmet need is recognized by the orphan drug designation we received today for our pharmaceutical CBD," said Michael Babich, president and chief executive officer. "Our pharmaceutical CBD is an alternative to plant derived cannabinoids, one which we believe will provide significant medical benefits and better address the unmet needs of patients across multiple indications including Dravet syndrome. We expect to file an Investigational New Drug Application (IND) for CBD in the second half of 2014."
Insys has more than seven years of research and development experience in the pharmaceutical cannabinoid space. The company manufactures pharmaceutical dronabinol (THC) and pharmaceutical CBD, both of which are cannabinoids, at its FDA-inspected and Drug Enforcement Administration (DEA) approved facility in Round Rock, Texas. Insys believes it is the only U.S.-based company with the capacity to produce pharmaceutical cannabinoids in scalable quantities.
In addition to Dravet syndrome, Insys plans to develop treatments for another rare form of pediatric epilepsy, Lennox-Gastaut syndrome, for which its pharmaceutical CBD recently received orphan drug designation. Insys is also evaluating the potential use of pharmaceutical CBD in several additional indications, including: adult epilepsy; chemotherapy-induced peripheral neuropathy; addiction in cocaine, heroin and opioids; and glioblastoma. Insys intends to pursue orphan drug designation for other indications that may qualify.
Orphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity, as well as certain financial incentives that can help support its development.
About Pharmaceutical CBD Cannabidiol (CBD) is one of at least 60 active cannabinoids identified in cannabis. CBD is considered to have a wider scope of medical applications than tetrahydrocannabinol (THC), and appears to have anti-convulsive action in animal models.
Insys' pharmaceutical CBD is over 99.5% pure cannabidiol. It is synthesized to be identical to the CBD extracted from cannabis, and is produced in a controlled environment. The FDA issued Drug Master File (DMF) #28255 to Insys for its CBD active pharmaceutical ingredient in May 2014.
Date: July 2, 2014