The US FDA has approved GW’s Epidiolex®, a cannabidiol (CBD) oral solution for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome – two rare, severe forms of epilepsy. This marks both the first FDA approval of a drug derived from the Cannabis sativa plant and the first drug to treat patients with Dravet syndrome.
CBD is a chemical derived from the plant C.sativa, more commonly known as marijuana; however, this is not the component that causes the intoxication associated with recreational use of the plant. Tetrahydrocannabinol is the primary psychoactive component of C.satvia.
"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"
“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,” commented FDA Commissioner Scott Gottlieb.
“Controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s drug approval process, is the most appropriate way to bring marijuana-derived treatments to patients. Because of the adequate and well-controlled clinical studies that supported this approval, prescribers can have confidence in the drug’s uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes,” Gottlieb added.
"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"
Lennox-Gastaut syndrome is characterized by multiple types of seizures that begin in early childhood, with more than three-quarters of affected individuals experiencing tonic seizures. Dravet syndrome also appears during the first year of life, and is associated with fever-related and myoclonic seizures.
“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,” remarked Billy Dunn, director of the Division of Neurology Products at the 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.”
The effectiveness of the CBD, Epidiolex, was studied in three randomized, double-blind, placebo-controlled trials involving 516 patients with either Dravet or Lennox-Gastaut syndrome. When taken alongside other medications, Epidiolex was demonstrated to effectively reduce the frequency of seizures, compared with the placebo.
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Common associated side-effects revealed in the trial include: sleepiness, sedation and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue; malaise and weakness; insomnia, sleep disorder and poor quality sleep; and infections, while more severe side effects such as suicidal thoughts, depression and agitation were also noted.
CBD remains a Schedule I substance under the Controlled Substances Act due to it being a chemical component of C.satvia, and has been evaluated in clinical and nonclinical studies to assess the potential for abuse of the drug.
The application was granted Priority Review designation by the FDA, and a Fast-Track designation was granted for the use of Epidiolex to treat Dravet Syndrome. Furthermore, Orphan Drug designation has been grated for both Lennox-Gastaut and Dravet syndromes.