The long and complex saga of cannabidiol (CBD) in the United States just crossed a major milestone. The US Food and Drug Administration (FDA) recently announced the approval of Epidiolex, a plant-derived CBD medicine, for treating seizure disorders. An FDA advisory panel recommended the drug for approval back in April, and the agency’s deadline for a decision was this week.
Epidiolex, manufactured by CW Pharmaceuticals, is approved for patients aged 2 and older, to treat two types of epileptic syndromes. Both disorders, Dravet syndrome and Lennox-Gastaut syndrome, often affect young children, and many patients suffer frequent debilitating seizures. Clinical trials have shown Epidiolex to be a promising treatment, particularly for the many patients who have not had success with other currently available treatments in controlling their seizures.
In a statement announcing the decision, FDA Commissioner Scott Gottlieb, M.D., touted the scientific rigor of the trials that led the agency to approve the drug, but was careful to note that it does not signify a wholesale endorsement of cannabis.
“This product approval demonstrates that advancing sound scientific research to investigate ingredients derived from marijuana can lead to important therapies. This new treatment provides new options for patients.
“This is an important medical advance. But it’s also important to note that this is not an approval of marijuana or all of its components. This is the approval of one specific CBD medication for a specific use. And it was based on well-controlled clinical trials evaluating the use of this compound in the treatment of a specific condition. Moreover, this is a purified form of CBD. It’s being delivered to patients in a reliable dosage form and through a reproducible route of delivery to ensure that patients derive the anticipated benefits. This is how sound medical science is advanced.”
Though the FDA’s approval of the drug is only for the two specific epileptic syndromes, it’s not uncommon for doctors to prescribe drugs for uses other than those approved by the FDA (off-label use). And even given the limited scope of the approval, the decision is still historic because it marks the first time the agency has approved a plant-derived cannabis medicine. In the past synthetic cannabinoid drugs have been available with a prescription, but Epidiolex is the first approved drug that is made from actual cannabis plants.
“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,” said GW Chief Executive Officer Justin Gover in a press release. “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.”
Speaking to CNN, Gover said the drug should be available in the fall of this year. He didn’t discuss pricing, but indicated that it would be announced soon following discussion with insurance companies. GW is also conducting a phase three clinical trial with the drug for another seizure-related condition called tuberous sclerosis complex, and will apply for supplemental approval for that condition if the results are promising. Additionally, Epidiolex has been submitted for approval to the European Medical Society, which is expected to announce a decision early next year.