The US Food and Drug Administration (FDA) has given approval for studies to begin on a medicinal form of marijuana for the treatment of intractable epilepsy in children.
The drug, cannabidiol (Epidiolex, GW Pharmaceuticals) is the largest nonpsychoactive component of the cannabis plant. The product has also been granted orphan drug status for the treatment of children with Dravet syndrome, a rare and severe syndrome of infantile-onset, genetic, drug-resistant epilepsy.
One of the investigators in the study, Orrin Devinksy, MD, New York University School of Medicine, New York, told Medscape Medical News that this is the first formal study of cannabidiol, although there have been anecdotal reports of “miraculous” benefits with similar products in severe epilepsy. Many of these reports have been featured as part of media coverage profiling parents lobbying to get access to this treatment for their children.