On Monday, the Food and Drug Administration (FDA) approved Epidiolex (cannabidiol) [CBD] 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. This is the first FDA-approved drug that contains a “purified drug substance derived from marijuana,“ as well as the first to be used in the treatment of Dravet syndrome.
CBD 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.
“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."Dr. Scott Gottlieb
“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 Dr. Scott Gottlieb. "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."
Dravet syndrome is a rare genetic condition that appears in the first year of life with frequent fever-related seizures. Later, several types of seizures develop and a potentially life-threatening state of continuous seizure activity requiring emergency medical care may occur. Lennox-Gastaut syndrome begins in childhood and is characterized by multiple types of seizures. Children have frequent seizures between the ages of 3 and 5, and most children develop learning and intellectual disabilities and require everyday assistance.
"We'll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products"Dr. Scott Gottlieb
Epidiolex was studied in three randomized, double blind, placebo-controlled clinical trials comprised of 516 patients with one of the two seizure disorders. Taken along with other medications, Epidiolex was shown to be effective in reducing the frequency of seizures when compared to placebo. According to the FDA, the most common side effects of the drug included drowsiness, lethargy, elevated liver enzymes and liver injuries, decreased appetite, diarrhea, rash, insomnia, malaise and infections. The most serious side effects of Epidiolex, which are true for all drugs that treat epilepsy, include suicidal thoughts, agitation, depression, aggression and panic attacks.
"We'll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers who are interested in bringing patients safe and effective, high quality products," Gottlieb said. "But, at the same time, we are prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims. Marketing unapproved products, with uncertain dosages and formulations can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases."
Source: Food and Drug Administration