On June 25, 2018, the U.S. Food and Drug Administration approved Epidiolex (cannabidiol) oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy (namely Lennox-Gastaut syndrome and Dravet syndrome) in patients two years of age and older28. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana. It is also the first FDA approval of a drug for the treatment of patients with Dravet syndrome.
The EPIDIOLEX clinical development program included three randomized, controlled Phase 3 clinical trials and an open-label extension study. Epidiolex's effectiveness was studied in three randomized, double-blind, placebocontrolled clinical trials involving 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex, taken along with other medications, has shown to be effective in reducing the frequency of seizures when compared with placebo.
The FDA granted Priority Review designation for this application. Fast-Track designation was granted for Dravet syndrome. Orphan Drug designation was granted for both the Dravet syndrome and Lennox-Gastaut syndrome indications. Outside the U.S., this medicine is currently under review by the European Medicines Agency (EMA) for the treatment of seizures associated with LGS and Dravet Syndrome. An EMA decision on whether to recommend approval is expected in the first quarter of 2019.
The FDA granted approval of Epidiolex to GW Research Ltd. EPIDIOLEX will be marketed in the U.S. by Greenwich Biosciences, the U.S. subsidiary of GW Pharmaceuticals plc29.
About EPIDIOLEX
EPIDIOLEX, the first prescription, plant-derived cannabinoid medicine in the United States and the first in a new class of anti-epileptic medications, is a pharmaceutical formulation of pure cannabidiol (CBD), is now FDAapproved for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in patients two years of age or older. Cannabidiol is a chemical component of the Cannabis sativa plant, more commonly known as marijuana. However, CBD does not cause intoxication or euphoria (the "high") that comes from tetrahydrocannabinol (THC). It is THC (and not CBD) that is the primary psychoactive component of marijuana. Under the Controlled Substances Act (CSA), CBD is currently a Schedule I substance because it is a chemical component of the cannabis plant. In support of this application, the company conducted nonclinical and clinical studies to assess the abuse potential of CBD, and has provided recommendations to the Drug Enforcement Administration (DEA) regarding controls under the CSA. DEA is required to make a scheduling determination.
About Dravet Syndrome
Dravet syndrome is a rare genetic condition that appears during the first year of life with frequent fever-related seizures (febrile seizures). Later, other types of seizures typically arise, including myoclonic seizures (involuntary muscle spasms). Additionally, status epilepticus, a potentially life-threatening state of continuous seizure activity requiring emergency medical care, may occur. Children with Dravet syndrome typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others.
About Lennox-Gastaut syndrome
Lennox-Gastaut syndrome begins in childhood. It is characterized by multiple types of seizures. People with Lennox-Gastaut syndrome begin having frequent seizures in early childhood, usually between ages 3 and 5. More than three-quarters of affected individuals have tonic seizures, which cause the muscles to contract uncontrollably. Almost all children with Lennox-Gastaut syndrome develop learning problems and intellectual disability. Many also have delayed development of motor skills such as sitting and crawling. Most people with Lennox-Gastaut syndrome require help with usual activities of daily living.
Footnotes
28. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm611046.htm
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