The U.S. Food and Drug Administration today approved the use of a syrup made with cannabidiol, which is derived from cannabis, to treat two forms of epilepsy.
It’s the first FDA-approved drug that contains a purified drug substance derived from marijuana. The drug would still need a new clearance from the Drug Enforcement Administration to be legal under federal law, but that is expected.
The FDA said in a press release Monday that it has approved Epidiolex, an oral CBD solution, for the treatment of seizures caused by Lennox-Gastaut syndrome and Dravet syndrome for patients over two years old. It’s the first time anything has ever been approved for use against Dravet syndrome, which has previously been essentially untreatable.
Epidiolex is a highly purified form of CBD, which comes from the Cannabis sativa plant, known to many who follow medical marijuana as the CBD oils that are gaining popularity in health and wellness circles. CBD doesn’t include THC, so it doesn’t produce a “high.”
Cannabis is currently classed as a Schedule I narcotic, which makes it federally illegal. But Schedule 1 drugs are considered to have no accepted medical use. Based on the FDA approval, CBD now could be considered to have that. The drug’s developers say they expect the DEA to reclassify CBD by the fall.
Epidiolex is made by British drug maker GW Pharmaceuticals.
“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, GW’s Chief Executive Officer said in a statement released by the company. “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.
“These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care,” Gover said.
“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,” said FDA Commissioner Scott Gottlieb. “And, the FDA is committed to this kind of careful scientific research and drug development.
“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. 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.”
The two conditions for which Epidiolex was approved are particularly bad, in part because of their severity, and in part because they’ve left doctors at a loss when trying to treat them.
“LGS and Dravet syndrome are two of the most severe and difficult-to-treat forms of childhood-onset epilepsy,” said Elizabeth Thiele, M.D., director of the pediatric epilepsy program at Massachusetts General Hospital, who was a lead researcher in the clinical program that tested the therapy. “These children and their families face a long and challenging road and very few achieve adequate seizure control.”
FDA Commissioner Gottlieb warned however, that the approval of one drug doesn’t necessarily make all marijuana-derived substances OK in the eyes of the agency.
“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,” he 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.”