On April 19, 2018, the Peripheral and Central Nervous System Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) unanimously recommended that the agency approve a new medicine derived from marijuana to treat seizures associated with two forms of epilepsy in children–Lennox-Gastaut syndrome and Dravet syndrome. The drug Epidiolex, manufactured by GW Pharmaceuticals, contains cannabidiol (CBD), which is a component of marijuana and is structurally unrelated to other drugs approved for the treatment of seizures.
The advisory committee concluded that the results from three adequate and well-controlled trials in Lennox-Gastaut syndrome and Dravet syndrome provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome. In general, the risks associated with CBD treatment appear acceptable, particularly given the findings of clinical efficacy in Lennox-Gastaut syndrome and Dravet syndrome, which are serious, debilitating, and life-threatening disorders. Although there is risk of liver injury with CBD, the committee concluded that the observed risk could be appropriately managed with inclusion of relevant language in labeling, education of prescribers regarding the risk of transaminase elevation and need for monitoring of liver enzyme levels, and further characterization of the risk in the post-market setting.
The recommendations of the advisory panel are not binding, and the FDA plans to make a decision in June.
- Panel recommends FDA approval of epilepsy drug derived from marijuana (STAT)
- FDA Briefing document: Cannabidiol (FDA)
- Devinsky O, Cross JH, Laux L, et al. Trial of cannabidiol for drug-resistant seizures in the Dravey Syndrome. New England Journal of Medicine. 2017;376:2011-2020.