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PHOENIX, AZ--(Marketwired - Jul 2, 2014) - Insys Therapeutics, Inc. (NASDAQ: INSY), 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 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 L. 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.

About Dravet Syndrome
Dravet syndrome, also known as Severe Myoclonic Epilepsy of Infancy (SMEI), is a rare and catastrophic form of intractable epilepsy that begins in infancy. Initial seizures are most often prolonged events and in the second year of life other seizure types begin to emerge. Development remains on track initially, with plateaus and a progressive decline typically beginning in the second year of life. Individuals with Dravet syndrome face a higher incidence of SUDEP (sudden unexplained death in epilepsy) and have associated conditions, which also need to be properly treated and managed.

About Insys Therapeutics, Inc.
Insys Therapeutics is a specialty pharmaceutical company that develops and commercializes innovative drugs and novel drug delivery systems of therapeutic molecules that improve the quality of life of patients. Using its proprietary sublingual spray technology and its capability to develop pharmaceutical cannabinoids, the company addresses the clinical shortcomings of existing commercial products. The company currently markets two products, Subsys, which is sublingual Fentanyl spray for break through cancer pain, and a generic version of Dronabinol (THC) capsules. The company plans to file a New Drug Application (NDA) for an oral liquid formulation of Dronabinol in the second half of 2014 and believes it is a clinically superior product to current Dronabinol capsules. The company is developing a pipeline of sublingual sprays, as well as pharmaceutical CBD.

Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding our expectations related to filing at least one Investigational New Drug application with the Food and Drug Administration in the second half of 2014 for pharmaceutical CBD, our plan to pursue the development of treatments for Lennox-Gastaut syndrome (LGS) and Dravet syndrome related to epilepsy, the potential benefits of our pharmaceutical CBD therapies to patients and our intention to pursue orphan drug designation for other indications that may qualify for orphan drug status. These forward-looking statements are based on management's expectations and assumptions as of the date of this press release, and actual results may differ materially from those in these forward-looking statements as a result of various factors, many of which are beyond our control. For a description of these risks facing the company, please see the risk factors described in our filings with the United States Securities and Exchange Commission, including those factors discussed under the caption "Risk Factors" in our Annual Report on Form 10-K for the year ended December 31, 2013 and any subsequent updates that may occur in our Quarterly Reports on Form 10-Q. Forward-looking statements speak only as of the date of this press release and we undertake no obligation to update or revise these statements, except as may be required by law.

 

 

CURE For questions, please contact the CURE office, 312.255.1801, or email info@cureepilepsy.org.

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