FDA Approves First Drug Made from Active Ingredient from Marijuana for Severe Epilepsy

FDA Approves First Drug Made from Active Ingredient from Marijuana for Severe Epilepsy

The FDA has approved Epidiolex (cannabidiol) oral solution for the treatment of seizures associated with two rare and severe forms of epilepsy, Lennox-Gastaut syndrome and Dravet syndrome. This is the first FDA-approved drug that contains a purified drug substance derived from marijuana.

Cannabidiol (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), the primary psychoactive component of marijuana.

FDA Commissioner Scott Gottlieb, M.D. stated “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… We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and work with product developers.

Dravet syndrome and Lennox-Gastaut syndrome are genetic conditions that appear early on in children. 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. 

Common side effects of Epidiolex 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. Like all epilepsy drugs, the most serious risks include thoughts about suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression and panic attacks. Epidiolex also caused liver injury, generally mild, but raising the possibility of rare, but more severe injury. More severe liver injury can cause nausea, vomiting, abdominal pain, fatigue, anorexia, jaundice and/or dark urine.

For further information, read the FDA News Release.