Today's approval "serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies". "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".
The drug is derived from the cannabidiol of cannabis, and, as the plant is yet to be decriminalized by federal regulators, its approval gives GW Pharma a lead among cannabis-exposed companies in the US financial markets. CBD is one of more than 100 chemicals found in marijuana. GW Pharmaceuticals grows the plants in the United Kingdom. The drug reduced seizures by half in 40 percent of Dravet patients participating in the trials. "If the FDA makes it clear that a cannabis-based pharmaceutical drug has medicinal value, then Congress can no longer claim there is no medicinal quality for cannabis". 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. He would not give any information on cost, saying only that it will be discussed with insurance companies and announced later. Epidiolex had previously received Fast Track Designation and Orphan Drug Status from the FDA.
Most patients with LGS and Dravet syndrome require multiple seizure medications and the majority are resistant to now approved anti-epileptic drugs. The chemical's use in seizure prevention is well-documented in reputable research, and now, after conducting its own trials, the FDA is on board.
Relying on homemade oil "is is a awful system, creating products that likely have more variability and can potentially contribute to more side effects and less consistency", Devinsky said. But Garris highlighted that numerous side effects occur when it is taken with other medications, which she said is a concern because most patients are on other medications. There was some concern about the drug's effects on the liver, but experts have said this risk could be addressed by doctors as they monitor their patients during treatment.
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.
A phase three clinical trial is underway for a third seizure-related condition called tuberous sclerosis complex, which begins in infancy and causes a sudden stiffening of the body, arms and legs, with the head bent forward. GW won't be able to market Epidiolex until the DEA reclassifies CBD.
As part of the FDA's review of the medication, the potential for abuse was assessed and found to be low to negative, according to Gover.
Despite today's approval, the FDA acknowledged in a statement that the cannabidinoid CBD still remains classified at this time as a schedule I controlled substance, and that the agency is "prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims".