Recently, the New England Journal of Medicine (NEJM)1 published the results of a Phase 3 study of a purified cannabidiol (CBD) medication, called Epidiolex, in Dravet syndrome. The process used in this study is considered the “gold standard” method of determining if a medication of any kind is safe and useful.
“An important strength of this trial is that it was conducted as a randomized, double-blind, controlled clinical trial, which is the gold-standard methodology for determining the safety and efficacy of a potential new therapeutic,” said Brandy Fureman, vice president of research and new therapies for the Epilepsy Foundation. “Before publication of this trial, much of the clinical evidence about CBD's effects on people's seizures was uncontrolled and anecdotal.”
The company conducting the trial, Greenwich Biosciences (formerly called GW Pharmaceuticals), has taken a traditional drug development approach with this compound and are attempting to gain U.S. Food and Drug Administration (FDA) approval for Epidiolex. Currently, there are no FDA approved treatments for Dravet syndrome.