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) for the treatment of glioma, which comprises approximately eighty percent of all malignant brain tumors. The ODD includes pontine glioma (PG), a devastating form of primary brainstem glioma.
“We are pleased to have received broad orphan drug designation for CBD to treat glioma,” said Michael L. Babich, President and Chief Executive Officer. “We will most likely focus initially on pontine glioma, or PG, which has multiple similarities with glioblastoma multiforme, for which our pharmaceutical CBD was granted ODD last month. We believe that this product has excellent potential as treatment for PG, and look forward to advancing its development and offering a potential efficacious treatment for patients.”
Insys, which has more than seven years of research and development experience in the pharmaceutical cannabinoid space, 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 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 glioblastoma multiforme (GBM), the most common and most aggressive malignant primary brain tumor in humans, and two rare forms of epilepsy, Lennox-Gastaut Syndrome and Dravet Syndrome. 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.
About Glioma A glioma is a type of tumor that arises from the glial cells of the brain or, less commonly, the spine. Gliomas comprise approximately 30% of all brain and central nervous system tumors and 80% of all malignant brain tumors.
About Pontine Glioma A pontine glioma (PG) is a glioma that arises along an area of the brain stem called the pons. PGs affect the cranial nerves, causing symptoms related to the nerves that supply the muscles of the eye and face, and muscles involved in swallowing. These symptoms include double vision, inability to close the eyelids completely, dropping one side of the face, and difficulty chewing and swallowing. The tumor also affects the “long tracks” of the brain, with resultant weakness of the arms or legs and difficulty with speech and walking. Symptoms usually worsen rapidly because the tumor is rapidly growing. These tumors have a poorer prognosis than the less common midbrain and medullary gliomas; there is no cure for PG, and currently no FDA approved treatments exist for PG.
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(R), which is sublingual Fentanyl spray for breakthrough cancer pain, and a generic version of Dronabinol (THC) capsules. The company submitted a New Drug Application (NDA) to the FDA for an oral liquid formulation of Dronabinol in August 2014 and believes the oral solution formulation has distinct advantages over the current formulation of dronabinol in a soft gel capsule. The company is developing a pipeline of sublingual sprays, as well as pharmaceutical cannabidiol.
Subsys(R) is a registered trademark of Insys Therapeutics, Inc.
Forward-Looking Statements This press release contains forward-looking statements, including statements regarding the development of Insys’ pipeline of sublingual sprays and pharmaceutical cannabinoids, the ability of Insys to advance development of a product related to CBD for glioma, and offering a potential cure for patients, Insys’ belief that it is the only U.S.-based company with the capacity to produce pharmaceutical cannabinoids in scalable quantities, and Insys’ belief that its oral solution formulation has distinct advantages over the current formulation of dronabinol in a soft gel capsule. 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.
SOURCE: Insys Therapeutics