On August 25, 2014 Insys Therapeutics reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to its pharmaceutical cannabidiol (CBD) for the treatment of glioblastoma multiforme (GBM), the most common and most aggressive malignant primary brain tumor in humans (Press release Insys Therapeutics, AUG 25, 2014, View Source [SID:1234500716]).

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"We are pleased to have received orphan drug designation for this aggressive and often incurable form of brain cancer. We look forward to advancing development of this product and offering a potential efficacious treatment for patients," said Michael L. Babich, President and Chief Executive Officer.

In addition to receiving ODD, Insys has recently entered into an exclusive licensing agreement with California Pacific Medical Center on behalf of its Research Institute (CPMCRI) based in San Francisco to license CPMCRI’s patent rights related to the usage of cannabinoids for the treatment of GBM.

In concert with the aforementioned exclusive licensing agreement, Insys is collaborating with Dr. Sean McAllister at CPMCRI with respect to his pre-clinical research focusing on the ability of CBD to sensitize GBM to current standard of care chemotherapy treatment. "Based on previous research conducted with CBD to treat brain tumors, we believe that there is supportive evidence for the use of CBD as an adjunct treatment in GBM and eagerly anticipate the results from our in-vivo models to further support clinical studies in humans," said Dr. McAllister.

Insys, which has more than seven years of research and development experience in the pharmaceutical cannabinoid space, manufactures pharmaceutical dronabinol (THC) and pharmaceutical CBD, both of which are cannabinoids, at its FDA-inspected and Drug Enforcement Administration (DEA) approved facility, located in Round Rock, Texas. The company recently submitted a Drug Master File (DMF #28255) for its CBD active pharmaceutical ingredient and believes that it is the only U.S.-based company with the capacity to produce pharmaceutical cannabinoids in scalable quantities.

Insys was previously granted ODD to its pharmaceutical CBD for the treatment of Lennox-Gastaut Syndrome and Dravet Syndrome, both rare forms of epilepsy. Insys is also evaluating 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 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.