The U.S. Food and Drug Administration (FDA) recently approved the drug Epidiolex, which contains cannabidiol (CBD), as an oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy—Lennox-Gastaut syndrome and Dravet syndrome—in patients two years of age and older (1). This marks the first FDA-approved drug that contains a purified drug substance derived from cannabis. It is also the first FDA approval of a drug for the treatment of patients with Dravet syndrome. The approval of Epidiolex was granted to GW Research Ltd.
“This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies. And, the FDA is committed to this kind of careful scientific research and drug development,” said FDA Commissioner Scott Gottlieb, M.D.
According to the FDA’s news release (1), 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.
The other condition Epidiolex was approved for, Lennox-Gastaut syndrome, also 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.
“The difficult-to-control seizures that patients with Dravet syndrome and Lennox-Gastaut syndrome experience have a profound impact on these patients’ quality of life,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “In addition to another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide a significant and needed improvement in the therapeutic approach to caring for people with this condition.”
Epidiolex’s effectiveness was studied in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. Epidiolex, taken along with other medications, was shown to be effective in reducing the frequency of seizures when compared with placebo. The most common side effects that occurred in Epidiolex-treated patients in the clinical trials were sleepiness, sedation, and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue, malaise, and weakness; insomnia, sleep disorder, and poor quality sleep; and infections.
For more information, please see the full news release from the FDA (1).