Insys' CBD Receives Orphan Status From FDA In Dravet Syndrome

Specialty pharmaceutical company Insys Therapeutics announced that its pharmaceutical cannabidiol (CBD) has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of rare pediatric-onset epilepsy, known as Dravet syndrome.

Cannabidiol (CBD) is a synthesized CBD extract identical to that from cannabis. It is over 99.5 percent pure cannabidiol and is grown in a controlled environment. CBD is only one of at least 60 active cannabinoids identified in cannabis and is thought to be more medically beneficial than tetrahydrocannabinol (THC). The company said data suggest that CBD has anti-convulsive effects in animal models.

Michael Babich, president and CEO of Insys, 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…. We expect to file an Investigational New Drug Application (IND) for CBD in the second half of 2014.” Babich also said that Insys’ CBD candidate, issued an FDA Drug Master File (DMF) as an active pharmaceutical ingredient last May, is a viable substitute to plant-derived cannabinoids for medical needs.

Dravet Syndrome is a rare form of intractable epilepsy that sets in at infancy. Also known as Severe Myoclonic Epilepsy of Infancy (SMEI), the condition shows itself during infancy; the infant presents with seizures that develop into prolonged events. Starting in the patient’s second year of life, new forms of seizures begin to develop. Patients with Dravet Syndrome suffer from a higher incidence of sudden unexplained death in epilepsy (SUDEP) and other associated conditions.

Last month, the company reported that its pharmaceutical CBD has also received Orphan Drug status from the FDA for a separate indication in another rare epilepsy form, the Lennox-Gastaut Syndrome. In addition, the company is considering CBD’s potential in other indications, such as adult epilepsy, chemotherapy-induced peripheral neuropathy, glioblastoma, and addiction to cocaine, heroin, and opioids.