Epidiolex contains CBD, which does not produce the "high" associated with marijuana.
The European Medicines Agency is now reviewing Epidiolex for treating seizures associated with LGS and Dravet Syndrome, with a decision on whether or not to recommend approval expected early next year. "And, the FDA is committed to this kind of careful scientific research and drug development", said Gottlieb. Shinnar said he will welcome approval of a medicine that had undergone stringent FDA review for safety and effectiveness.
And Epidiolex's approval doesn't mean other CBD substances are OK to sell, Gottlieb said. CBD is one of more than 100 chemicals found in marijuana. The drug contains only trace amounts of the psychoactive element THC and does not induce euphoria.
He added, "This is how sound medical science is advanced".
There are side effects, the most common being sleepiness, Gover said. "In the artisanal products there's often a huge variation in doses from bottle to bottle depending on where you get it". The drugs are listed as Schedule II and Schedule III, meaning they have medicinal value but also potential for abuse. The Figi family, whose daughter Charlotte was diagnosed with Dravet syndrome at 3 months old, has been extremely vocal about medical marijuana's merits. But then Hendershot learned about a trial of Epidiolex at New York University.
It's an option for those patients who have not responded to other treatments to control seizures. As well, CBD remains in a legal grey area, making it hard for patients outside of legal states to acquire.
The immediate impact of Monday's approval on these products is unclear.
"Marketing unapproved products, with uncertain dosages and formulations, can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases", he said.
Most CBD producers sidestep the issue by making only broad claims about general health and well-being.
The FDA seal is nonetheless seen to be a boon for the broader industry.
GW Pharmaceuticals grows the plants in the United Kingdom. Wall Street analysts have previously predicted it could cost $25,000 per year, with annual sales eventually reaching $1 billion.
The drug, administered as an oil, could significantly improve the quality of life for patients with two types of childhood epilepsy: Dravet syndrome, a genetic illness that kills up to 20 percent of sufferers before age 20, and Lennox-Gastaut syndrome, a form that begins between ages 3 and 5 and causes intellectual disability.
Nine states and the District of Columbia have legalized marijuana for recreational use.
Despite increasing acceptance, there is little rigorous research on the benefits and harms of marijuana.
Epidiolex will be marketed by Greenwich Biosciences, a subsidiary of GW Pharmaceuticals.
Prior to the FDA decision, a press officer for the DEA confirmed to Leafly that the FDA decision will prompt action from the DEA.
With the historic approval, the London-based company behind the drug, GW Pharmaceuticals plc, is expecting another consequential decision in the coming weeks: getting the US Drug Enforcement Administration to reclassify CBD. And the company plans to continue importing the medicine, bypassing onerous US regulations on manufacturing restricted substances.
The European Medicines Agency is expected to rule on the therapy in early 2019.