In an unprecedented move, the Food and Drug Administration (FDA) has approved Epidiolex—the first pharmaceutical drug made from cannabis (marijuana)—for the treatment of two types of epilepsy.
Epidiolex is specifically approved to treat Dravet syndrome and Lennox-Gastaut syndrome, two rare and severe forms of epilepsy that tend to begin in childhood.
But there's a possibility that doctor's may prescribe Epidiolex for other forms of epilepsy off-label. The drug is made from cannabidiol (CBD), a non-psychoactive compound found in cannabis that doesn't produce the typical "high" feeling.
The drug's approval was based on three clinical trials that involved 516 patients with either of the syndromes (as well as some additional safety studies), which showed that patients who took Epidiolex experienced a decrease in the number of seizures they had compared to placebo. The most common side effects of Epidiolex included sleepiness, elevated liver enzymes, decreased appetite, diarrhea, rash, fatigue, insomnia, and infections.
“Today’s approval of Epidiolex is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat two severe, childhood-onset epilepsies,” Justin Gover, chief executive officer or GW Pharmaceuticals, said in a statement. “This approval is the culmination of GW’s many years of partnership with patients, their families, and physicians in the epilepsy community to develop a much needed, novel medicine.
Other drugs based on compounds in cannabis are already on the market, such as dronabinol (Marinol), a synthetic form of THC, the main component of cannabis, which is used to treat nausea and vomiting that may accompany chemotherapy. But Epidiolex is the first FDA-approved drug that's made directly from the cannabis plant (grown at a facility in the U.K.), rather than created synthetically.
Under current federal law, cannabis and anything derived from the plant (including CBD) is considered a Schedule I drug. But the federal approval of a CBD-based drug challenges that classification head on.
A Schedule I classification means that, according to the federal government, there's a high potential for abuse and no accepted medical use for the drug.
"Under the Controlled Substances Act (CSA), CBD is currently a Schedule I substance because it is a chemical component of the marijuana plant," the press release reads. "In support of this application, the company conducted nonclinical and clinical studies to assess the abuse potential of CBD."
Additionally, the press release explained that "the FDA prepares and transmits, through the U.S. Department of Health and Human Services, a medical and scientific analysis of substances subject to scheduling, like CBD, and provides recommendations to the Drug Enforcement Administration (DEA) regarding controls under the CSA." But it's up to the DEA to make the final scheduling decision.
At this time, it's "unclear whether the scheduling of Epidiolex per se will have any tangible impact on the scheduling of CBD," Paul Armentano, deputy director of NORML, tells SELF. But, "if the DEA is compelled to revisit the issue of CBD scheduling," he predicts that CBD present in a standardized, FDA-approved compound like Epidiolex may move into Schedule III, which indicates that the DEA believes they have a moderate to low potential for dependence and some medical use. (Drugs currently in Schedule III include ketamine, Tylenol with codeine, and anabolic steroids, to name a few examples.) But CBD present in an unapproved product would remain in Schedule I, Armentano predicts.
This kind of breakdown already exists in the case of dronabinol, which is a Schedule III drug, Armentano notes. But, of course, cannabis itself and THC are still in Schedule I.
The FDA also made it clear that it will continue to crack down on CBD products that are not FDA-approved.
CBD has become something of a trendy wellness product in recent years, which the FDA finds concerning because it is still considered a Schedule I drug and is often mislabeled or sold with promotional materials that overpromise it's supposed benefits.
The approval of Epidiolex "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, M.D., said in the press release.
"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," Dr. Gottlieb continued. "Marketing unapproved products, with uncertain dosages and formulations can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases.”