GW Pharma receives FDA orphan drug designation for cannaboid-based agent

UK-based GW Pharmaceuticals (Nasdaq: GWPH) has been granted orphan drug designation by the US Food and Drug Administration (FDA) for its investigational product candidate Epidiolex for use in treating children with Dravet syndrome.

Epidiolex contains plant-derived Cannabidiol (CBD) as its active ingredient and is an oral liquid formulation of a highly purified extract of CBD, a non-psychoactive molecule from the cannabis plant. Following receipt of this orphan designation, GW anticipates holding a pre-IND meeting with the FDA in the near future to discuss a development plan for Epidiolex in Dravet syndrome.

Stephen Wright, GW’s R&D director, said: “GW is proud to be at the forefront of this important new program to treat children with Dravet Syndrome and potentially other forms of intractable childhood epilepsy. For families in these circumstances, their lives are significantly impacted by constant and often times very severe seizures in children where all options to control these seizures have been exhausted. GW intends to advance a full clinical development program for Epidiolex in Dravet syndrome as quickly as possible, whilst at the same time helping families in the short term through supporting physician-led INDs to treat intractable cases.”