Epidiolex, a medicine that uses cannabidiol (CBD) as an active ingredient, has been designated an orphan drug for Dravet syndrome by the US Food and Drug Administration, giving manufacturer GW Pharmaceuticals the go-ahead to run a clinical development programme next year.

CBD is a non-psychoactive compound derived from the cannabis plant. In GW Pharmaceuticals’ formulation, it is administered orally, as a liquid.

The orphan drug designation means it is recognised as a potential therapy for a condition that only affects a small number of people – in this case a rare form of paediatric epilepsy.

...GW Pharmaceuticals expects to begin a formal clinical development programme for Epidiolex in 2014. In the meantime, some US paediatric epilepsy specialists will be able to treat patients with the drug immediately thanks to seven Investigational New Drug (IND) applications granted to the firm by the FDA.