Insys Gets FDA Orphan Drug Designation For Pharmaceutical Cannabidiol

(RTTNews) - Insys Therapeutics, Inc. (INSY), a specialty pharmaceutical company developing innovative drugs and novel drug delivery systems, Wednesday announced that the U.S. Food and Drug Administration or FDA has granted orphan drug designation to its pharmaceutical cannabidiol or CBD for the treatment of Dravet syndrome, a rare pediatric-onset epilepsy.

The company said it expects to file an Investigational New Drug Application or IND for CBD in the second half of 2014.

Insys manufactures pharmaceutical dronabinol or THC and pharmaceutical CBD, both of which are cannabinoids, at its FDA-inspected and Drug Enforcement Administration or DEA approved facility in Round Rock, Texas.

The company said it believes that it is the only U.S.-based company with the capacity to produce pharmaceutical cannabinoids in scalable quantities.

Michael Babich, President and Chief Executive Officer, said, "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. 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."

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.

The company is also evaluating the potential use of pharmaceutical CBD in several additional indications.

Orphan drug designation is granted by the FDA Office of Orphan Products Development 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.