The FDA has approved Epidiolex — the first drug the agency has approved that consists of a purified drug substance derived from marijuana — for the treatment of seizures in patients aged 2 years and older with Dravet Syndrome and Lennox-Gastaut Syndrome, according to a press release.
Epidolex (cannabidiol, GW Research Ltd.) is not associated with the feeling of euphoria caused by tetrahydrocannabinol (THC), the release stated.
“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,” Billy Dunn, MD, director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research said in the release.
“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,” he added.
The release stated that cannabidiol’s approval came after three placebo-controlled, randomized, double-blind, trials involving 516 patients with either Lennox-Gastaut syndrome or Dravet syndrome. When taken with other drugs, the oral solution cannabidiol was effective in reducing the frequency of seizures vs. placebo.
Common adverse events in patients who received cannabidiol included elevated diarrhea; elevated liver enzymes; fatigue; infections; insomnia; poor quality sleep and sleep disorder; loss of appetite; malaise and weakness; sleepiness, sedation and lethargy. Patients who receive cannabidol will also get a Medication Guide that describes important information about the drug’s uses and risks, according to the release.
“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,” FDA Commissioner Scott Gottlieb, MD, said in the release.
“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. But, at the same time, we are prepared to take action when we see the illegal marketing of [cannabidiol]-containing products with serious, unproven medical claims,” he added.
Dunn and Gottlieb work for the FDA.