UPDATE: Insys Therapeutics Receives FDA Orphan Drug Designation For Its Pharmaceutical Cannabidiol As A Potential Treatment For Pediatric Schizophrenia

Insys Therapeutics, Inc.

, a specialty pharmaceutical company that is developing and commercializing innovative drugs and novel drug delivery systems, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its pharmaceutical cannabidiol (CBD) candidate for the treatment of pediatric schizophrenia. This marks the fifth ODD for the company's CBD program. "Receiving orphan drug designation for CBD to treat pediatric schizophrenia is a lifeline for the thousands of children living with this disorder, and the parents who care for them," said Michael L. Babich, President and Chief Executive Officer. "We are excited t o explore the potential utility of our CBD candidate for this severe disorder alongside the other indications for which it has been granted ODD. We look forward to dosing our first human subjects with CBD in a Phase I clinical trial, which we expect to take place in early 2015." Insys has previously been granted ODD for its pharmaceutical CBD for the treatment of glioma, a tumor of the brain or nervous system, glioblastoma multiforme (GBM), the most common and most aggressive malignant primary brain tumor in humans, and two rare forms of pediatric epilepsy, Lennox-Gastaut Syndrome and Dravet Syndrome. Insys intends to pursue orphan drug designation for its pharmaceutical CBD for the treatment of other indications that may qualify. 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 upon approval, as well as certain financial incentives that can help support its development. Insys continues to evaluate the potential use of pharmaceutical CBD in several additional indications, including adult epilepsy, chemotherapy-induced peripheral neuropathy, and addiction in cocaine, amphetamines and opioids. Insys manufactures pharmaceutical CBD and pharmaceutical dronabinol (THC), both of which are cannabinoids, at its FDA-inspected and Drug Enforcement Administration (DEA)-approved facility in Round Rock, Texas. The company has DEA approval to produce 15kg of CBD in 2014, and expects a larger quota in 2015 for its continued clinical studies. Insys believes that it is the only U.S.-based company with the capacity to produce pharmaceutical cannabinoids in scalable quantities.