Today, the US Food and Drug Administration (FDA) approved cannabidiol (Epidiolex) [CBD] oral solution in patients aged 2 years and older for the treatment of severe and rare types of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome.
“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, MD in a recent statement
, more commonly known as marijuana, is a chemical component of the Cannabis sativa plant. However, intoxication or euphoria (the “high”) that comes from tetrahydrocannabinol (THC) is not caused by the medicaiton.
“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,” added Dr Scott. “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.
In 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome, CBD’s effectiveness was evaluated in 3 randomized, double-blind, placebo-controlled clinical trials involving CBD taken in with other medications. Result showed CBD to be effective in reducing the frequency of seizures when compared with placebo.
“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,” furthered Dr Scott. “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.”
Sleepiness, sedation, and lethargy; elevated liver enzymes; decreased appetite; diarrhea; rash; fatigue, malaise and weakness; insomnia, sleep disorder and poor-quality sleep; and infections included the most common side effects that occurred in CBD-treated patients in the clinical trials.
Risks with CBD include: suicidal thoughts, suicide attempts, feelings of agitation, new or worsening depression, aggression and panic attacks in addition to liver injury, which can cause nausea, vomiting, abdominal pain, fatigue, anorexia, jaundice and/or dark urine.
“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,” added Billy Dunn, MD, 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.”