Epidolex, the first FDA-approved cannabis-based medication, is now available via prescription in all US states. Produced by GW Pharmaceuticals, the drug received approval by the US Food and Drug Administration (FDA) in June, followed by a narrow rescheduling by the US Drug Enforcement Administration (DEA), which categorized Epidolex as a Schedule V drug on the Controlled Substances Act (CSA). Epidiolex utilizes cannabidiol (CBD), a non-psychoactive cannabinoid found in the Cannabis sativa L. plant, as its active ingredient. “Marihuana” will continue to be listed as a Schedule I substance on the CSA.
Epidiolex is approved for the treatment of two rare epileptic seizure disorders (Dravet syndrome and Lennox-Gestaut syndrome, both of which appear in early childhood), but can also be prescribed “off-label” for other medical conditions as determined by a doctor. According to the US Department of Health and Human Services Agency for Healthcare Research and Quality, 1 in 5 prescriptions written in the US today are for off-label use.
Dr. Scott Gottlieb, the Commissioner of the FDA, issued a statement saying, “Adequate and well-controlled clinical studies supported Epidiolex’s approval, so 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 FDA will continue to support rigorous scientific research on the potential medical uses of marijuana-derived products and stand ready to work with product developers who are interested in bringing patients safe and effective, high quality products.”
Justin Gover, the CEO of GW Pharmaceuticals, said in a written statement, “Because these patients have historically not responded well to available seizure medications, there has been a dire need for new therapies that aim to reduce the frequency and impact of seizures. We are committed to ensuring that these patients can access this novel cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and is eligible to be covered by insurance for appropriate patients.”
Dr. Orrin Devinsky, director of the Comprehensive Epilepsy Center at NYU Langone Health, who oversaw two of the clinical trials of Epidiolex, told CNN, “The off-label use of CBD and Epidiolex will be a fascinating subject to look at over the coming years,” Devinsky said. “Many, many patients would like to have access to it who don’t fit the criteria, and I believe many doctors will try to prescribe it, as will I for some people who I think have tried many, many medications and for whom it might be beneficial.”
The price of Epidiolex has been set at roughly $32,500 annually. GW Pharmaceuticals spokesperson Stephen Schultz told Endpoints News, “Based on anticipated dosing and patient weight assumptions, we believe that this translates to a weighted average gross price in the first year of $32,500, which is in line with other branded anti-epileptic drugs used to treat these conditions.” He also told the publication that GW Pharmaceuticals is developing a patient support program to help lower out-of-pocket costs or to potentially provide Epidiolex free to eligible patients.