The Food and Drug Administration (FDA) last week approved Epidiolex (cannabidiol; GW Pharmaceuticals) for the treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome in patients 2 years of age and older. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana; it’s also the first FDA approval of a drug for the treatment of patients with Dravet syndrome.
Dravet syndrome and Lennox-Gastaut syndrome are two forms of treatment-resistant epilepsy. Dravet syndrome is a rare and severe form of infantile-onset epilepsy that typically appears during the first year of life in previously healthy infants; by two years of age, children with Dravet syndrome usually show signs of developmental delay. Seizures can regress in adulthood, but most patients have ongoing seizures that don’t adequately respond to medication. Lennox-Gastaut syndrome is another severe form of childhood-onset epilepsy that can include several different types of seizures. People with Lennox-Gastaut syndrome begin having frequent seizures in early childhood, usually between 2 and 7 years of age.
Cannabidiol, the active ingredient in Epidiolex, is a chemical component of the Cannabis sativa plant (marijuana); cannabidiol does not cause the intoxication or euphoria that comes from tetrahydrocannabinol (THC), another component of the Cannabis sativa plant. The FDA approval of Epidiolex was based on results from 3 randomized, double-blind, placebo-controlled clinical trials involving patients with either Lennox-Gastaut syndrome or Dravet syndrome. The initial FDA approval for Epidiolex is for a narrow indication, but the company is developing cannabidiol formulations for numerous other indications.
Cannabidiol is currently controlled in Schedule I under the Controlled Substances Act. Schedule I substances have no currently accepted medical use in the U.S., a high potential for abuse, and a lack of demonstrated safety for use. A scheduling recommendation from the FDA reflecting the Epidiolex approval has been submitted to the Drug Enforcement Administration (DEA). The manufacturer anticipates that cannabidiol will be reclassified by the DEA within a few months; this would open the door for development of additional cannabidiol-based products. Until the DEA decides to change cannabidiol to a different schedule, Epidiolex remains a Schedule I controlled substance and may not be marketed in the U.S.
The Hayes Technology Prognosis team has been following the development of Epidiolex since 2016. Hayes Technology Prognosis follows health technologies that are nearing regulatory approval as well as those that are newly approved; our focus is on high-impact, high-cost technologies that have the potential to change patient care practices. Each Prognosis Overview follows a single drug or device for a single indication; our overviews are updated as the technology moves through the product development cycle.
A Hayes technology Prognosis overview on Epidiolex is available here. We’ll continue to monitor this topic and update our report pending DEA action on cannabidiol.