Epidiolex filed for two rare forms of childhood-onset epilepsy.

GW Pharmaceuticals plc, a biopharmaceutical company focused on discovering, developing and commercializing novel therapeutics from its proprietary cannabinoid product platform, announced it has submitted its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for Epidiolex (cannabidiol or CBD) as adjunctive treatment for seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, two highly treatment-resistant forms of childhood-onset epilepsy. GW has received Orphan Designations from the EMA for Epidiolex for the treatment of LGS, Dravet syndrome, and recently, West syndrome and Tuberous Sclerosis Complex. In October 2017, GW completed the submission of its rolling Epidiolex New Drug Application (NDA) to the United States Food and Drug Administration (FDA), also for seizures associated with LGS and Dravet syndrome. This NDA has been accepted by the FDA for Priority Review.

The MAA for Epidiolex is supported by data from three Phase III safety and efficacy studies, each of which met their primary endpoint. Epidiolex was generally well tolerated in these trials. The MAA includes safety data on approximately 1,500 patients, with approximately 400 patients on continuous therapy for more than one year. Approximately 26% of the Epidiolex patients from the Phase III pivotal program came from European sites. Beyond pivotal safety and efficacy data, the MAA includes a comprehensive clinical pharmacology, pre-clinical and toxicology package.