FDA orphan status for Insys' cannabidiol as treatment for Dravet syndrome

US specialty pharma firm Insys Therapeutics (Nasdaq: INSY) yesterday revealed that the US Food and Drug Administration has granted orphan drug designation for its pharmaceutical cannabidiol (CBD) for the treatment of Dravet syndrome, a rare pediatric-onset epilepsy.

This follows swiftly on the  FDA decision last month to grant orphan status for the product for the treatment of Lennox-Gastaut syndrome, which is also 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 Babich, president and chief executive of Insys, adding: "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."