Orphan drug designation spurs trials for cannabidiol in Dravet syndrome

In a survey and other anecdotal reports, parents of children with drug-refractory epilepsy syndromes have reported successful treatment with cannabidiol-enriched marijuana. But little high-quality evidence is available about the efficacy and safety of cannabidiol in patients with Dravet, Lennox-Gastaux, and other pediatric epilepsy syndromes, as shown by a recent Cochrane Collaboration review of four randomized trials totaling 48 patients with epilepsy that found no adverse effects with 200-300 mg daily over short periods but did not report on the efficacy of the drug.