Epidiolex

ANALYST OUTLOOK

GW Pharmaceuticals is positioning its unique drug Epidiolex (cannabidiol) to target intractable childhood epilepsy syndromes with high unmet need. Epidiolex possesses distinct novelty as a first-in-class cannabinoid with little affinity to the cannabinoid receptor 1 (CB1)associated with psychoactivity. The drug is also exceptional in its demonstrated efficacy in treating Dravet syndrome, as there are currently no approved treatments in the US. The positive Phase III outcome for Dravet syndrome, supported by encouraging results from Epidiolex’s expanded access program (EAP), should facilitate the drug’s approval. Furthermore, Epidiolex has shown comparable efficacy in patients with Lennox-Gastaut syndrome (LGS) to other anticonvulsants approved for this indication. GW Pharmaceuticals is planning to submit a New Drug Application (NDA) in the US in mid-2017, and to follow this with a submission to the European Medicines Agency (EMA). The company is also investigating Epidiolex’s potential in Phase III trials in infantile spasms (IS) and tuberous sclerosis complex (TSC). Datamonitor Healthcare believes that further demonstrating efficacy in these indications will likely encourage physicians to perceive Epidiolex as the product of choice for the treatment of childhood intractable epilepsy.

DRUG OVERVIEW

GW Pharmaceuticals is developing Epidiolex, a first-in-class cannabinoid drug for the treatment of childhood seizures. The drug is thought to exert its anticonvulsant effects via the activation of several pathways, such as ion channel control, anti-inflammatory effects, endocannabinoid system modulation, and/or regulation of epilepsy-related genes, although this remains to be determined. Importantly, research indicates that Epidiolex has no or little binding affinity to the CB1, which is otherwise responsible for the unwanted psychoactivity associated with tetrahydrocannabinol (THC) use (GW Pharmaceuticals, 2016a).