For the first time, the US Food and Drug Administration has approved a drug made from cannabis. Epidiolex treats severe forms of childhood epilepsy — but the Drug Enforcement Administration will need to reclassify this form of cannabis before the drug can be put on the market. For almost half a century, the DEA has classified cannabis as a Schedule I drug, along with heroin and LSD. (For context, cocaine and meth are Schedule II drugs.) Schedule I drugs are considered to have “no currently accepted medical use and a high potential for abuse. “ As a result, it is extremely difficult for scientists to research the substance and pharmaceutical companies aren’t allowed to use it. But now the FDA has approved Epidiolex as a medical drug. Epidiolex contains cannabidiol, or CBD — a chemical that comes from the cannabis plant but does not have psychoactive effects — and is administered as an oil. According to DEA spokesperson Melvin Patterson, the administration had been considering reclassifying CBD and “the FDA’s findings on something like Epidiolex will weigh heavily on the decision-making process. Still, there’s no update on whether the approval will change the timeline of the proposed reclassification. The DEA could also reclassify CBD, but leave cannabis itself at Schedule I. Epidiolex treats two forms of epilepsy, Dravet syndrome and Lennox-Gastaut syndrome, which rarely respond to treatment. It is manufactured by GW Pharmaceuticals and clinical trials have shown that kids taking Epidiolex had nearly 40 percent fewer seizures per month. The scope of the drug is limited, and the approval is for “a single compound that comes from the plant and is approved for two very specific forms of epilepsy,” said Daniel Friedman, an associate professor of neurology at NYU Langone’s Comprehensive Epilepsy Center who co-authored a study investigating Epidiolex to treat Dravet syndrome. But doctors could prescribe it off-label for other forms of epilepsy, and FDA approval could be the domino effect that leads to DEA reclassification and a renaissance of cannabis research. We have reached out to GW Pharmaceuticals for comment and will update the post if the company responds.<doc-sep>The Food and Drug Administration approved the first cannabis-derived drug on Wednesday. Called Epidiolex, the GW Pharmaceuticals drug is designed to treat two rare forms of childhood epilepsy using a marijuana compound called CBD. Experts say the approval will galvanize research into other marijuana-based drugs and unleash a sea change for the CBD industry. A new drug derived from marijuana just became the first of its kind to get the green light from the US government. Called Epidiolex, the drug is designed to treat two rare forms of childhood epilepsy using a cannabis compound called cannabidiol (or CBD). British-based GW Pharmaceuticals makes the drug. It does not contain THC, the well-known psychoactive component of marijuana responsible for the drug's characteristic high. The federal thumbs-up comes on the heels of several months of promising research results and a positive preliminary vote from the Food and Drug Administration this spring. Experts are hopeful that the approval will unleash a wave of new interest in the potential medical applications of CBD and other marijuana compounds. “This approval serves as a reminder," Scott Gottlieb, the FDA commissioner, said in a statement on Monday, "that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies." In three large clinical trials which the FDA considered before giving Epidiolex the official green light, researchers presented strong evidence that the pharmaceutical-grade CBD in the medicine had the power to significantly curb some of the worst symptoms of two of the hardest-to-treat forms of epilepsy, known as Lennox-Gastaut syndrome and Dravet syndrome. "This is clearly a breakthrough drug for an awful disease," John Mendelson, a panel member and senior scientist at the Friends Research Institute, said at a public FDA meeting this spring that was called to discuss the scientific merits of the drug. But although the green light means that patients will soon be able to access Epidiolex with a doctor's prescription, many will also likely turn to less expensive sources of CBD, such as those sold in marijuana dispensaries. Researchers and advocates cautioned against this, however, with the caveat that it's impossible to verify that what's in those products is actually pharmaceutical-grade CBD. 'A boatload of evidence to show at this point' Courtesy GW Pharma Starting last spring with a study presented at the American Academy of Neurology's yearly meeting in Boston, GW Pharmaceuticals has consistently presented strong clinical studies that appear to support Epidiolex. "There’s a boatload of evidence to show for this drug at this point," Orrin Devinsky, a lead author on one of those studies and a neurologist at NYU Langone Health, told Business Insider. A month after the Neurology meeting, Devinksy and his colleagues published a positive study of the drug in children with Dravet syndrome in the New England Journal of Medicine. Roughly 43% of the children in that study who got the drug saw their number of seizures cut in half, and 5% stopped having seizures entirely. In comparison, children who got a placebo had barely any noticeable change in their symptoms. Almost exactly a year later, Devinksy and his team followed up those results with another favorable study in the same journal using a smaller dose of the drug in patients with Lennox-Gastaut. Devinsky said that low dose might be "the sweet spot" where most patients can achieve relief from symptoms without any unwanted side effects, such as drowsiness. Also, although Epidiolex is designed to treat only two types of epilepsy, medical professionals can technically prescribe it "off-label" for other conditions as well. (The anesthetic ketamine, for example, is prescribed this way for some hard-to-treat forms of depression.) "We would expect that once this is approved as a drug it's quite likely this will be tried in other populations off-label so it has a big opportunity to affect others," Lubbers said. Epidiolex vs. other CBD products Scientists and advocates representing families of patients with epilepsy are hailing Epidiolex's arrival as a long-awaited treatment. But they are also aware that desperate patients — especially parents of young children — may actively seek alternate sources of CBD that may be cheaper and don't require a doctor's prescription. In states where marijuana is legal (or in states with laws legalizing CBD on its own), oils and salves made with CBD are widely available. But experts caution that these products may not be what they seem. "What's different with [Epidiolex] is that this is a well-studied and well-controlled product," Laura Lubbers, the chief scientific officer of a nonprofit called Cure that funds epilepsy research, told Business Insider in April. But most dispensary-grade CBD products are not well-studied or well-controlled. For a 2017 study published in the Journal of the American Medical Association, researchers tested 84 products purchased from 31 different online CBD sellers. Roughly seven out of 10 items had different levels of CBD than what was written on the label. Of all of the items they tested, roughly half of the items had more CBD than was indicated; a quarter had less. And 18 of the samples tested positive for THC, despite it not being listed on the label. "The main thing is that CBD as approved by the FDA is pharmaceutical-grade CBD. It's manufactured under stringent standards, the same as other FDA-approved drugs," Shlomo Shinnar, the president of the American Epilepsy Society and a professor of neurology and epidemiology at the Albert Einstein College of Medicine, told Business Insider. "That’s not the same as when people tell you, 'Oh, I’ve got marijuana and it’s high in CBD,' or 'Oh, I've got a CBD product.'" A powerful catalyst for deeper research into cannabis-derived drugs An employee holds up cannabis in the laboratory at the headquarters of herbal medicines manufacturer Bionorica in Neumarkt, Germany February 9, 2018 REUTERS/Michaela Rehle Epidiolex's approval could also be a powerful catalyst for deeper research into other marijuana-derived medicines. Cannabis has more than 400 compounds, of which CBD and THC are only two, and researchers think the others could hold promise as well. The drug's green light also means that the Drug Enforcement Agency now has 90 days to reschedule CBD, which it listed in January of last year as a "marihuana extract" separate from "marihuana" or THC. That means instead of being listed alongside marijuana as a Schedule 1 drug with "no currently accepted medical use," CBD will soon be categorized as either a Schedule 2 or 3 drug, much like the popular ADHD medication Adderall. "We don't have a choice on that," DEA public affairs officer Barbara Carreno told Business Insider. "It absolutely has to become Schedule 2, 3, 4, or 5." Once that happens, it'll unleash what Carreno called a "sea change" for CBD manufacturers and the industry as a whole, which up until this point has existed in a legal grey area. Apart from CBD, researchers are also actively studying THC and other marijuana compounds for a range of potential medical uses, from relieving pain to soothing severe nausea. Although Epidiolex is the first marijuana plant-based drug to land FDA approval, the agency has already given the green light to drugs whose active ingredient is a lab-made version of THC, for example. In the meantime, experts look forward to seeing Epidiolex made available to patients in need. "I'm not currently aware of any other major new drugs that are close to being where this drug is," Shinnar said.<doc-sep>
Story highlights Epidiolex treats two types of epileptic syndromes The drug is expected to become available in the fall (CNN) — The US Food and Drug Administration approved a cannabis-based drug for the first time, the agency said Monday. Epidiolex was recommended for approval by an advisory committee in April, and the agency had until this week to make a decision. The twice-daily oral solution is approved for use in patients 2 and older to treat two types of epileptic syndromes: Dravet syndrome, a rare genetic dysfunction of the brain that begins in the first year of life, and Lennox-Gastaut syndrome, a form of epilepsy with multiple types of seizures that begin in early childhood, usually between 3 and 5. “This is an important medical advance,” FDA Commissioner Dr. Scott Gottlieb said in a statement Monday. “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 the “first pharmaceutical formulation of highly-purified, plant-based cannabidiol (CBD), a cannabinoid lacking the high associated with marijuana, and the first in a new category of anti-epileptic drugs,” according to a statement Monday from GW Pharmaceuticals, the UK-based biopharmaceutical company that makes Epidiolex. Cannabidiol is one of more than 80 active cannabinoid chemicals, yet unlike tetrahydrocannabinol, or THC, it does not produce a high. The FDA has approved synthetic versions of some cannabinoid chemicals found in the marijuana plant for other purposes, including cancer pain relief. Justin Gover, chief executive officer of GW Pharmaceuticals, described the approval in the statement as “a historic milestone.” He added that the drug offers families “the first and only FDA-approved cannabidiol medicine to treat two severe, childhood-onset epilepsies.” “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. Epidiolex will become available in the fall, Gover told CNN. He would not give any information on cost, saying only that it will be discussed with insurance companies and announced later. With Epidiolex meeting FDA standards, the drug will “finally be made available to the thousands that may benefit from it,” he said. It’s an option for those patients who have not responded to other treatments to control seizures. According to the Epilepsy Foundation, up to one-third of Americans who have epilepsy have found no therapies that will control their seizures. Shauna Garris, a pharmacist, pharmacy clinical specialist and adjunct assistant professor at the University of North Carolina’s Eshelman School of Pharmacy, said the drug is effective and works somewhere between “fairly” and “very well.” She has not used Epidiolex in her own clinical practice and was not involved in the development of the drug but said she’s not sure it will live up to “all of the hype” that has surrounded it. There are side effects, the most common being sleepiness, Gover said. But Garris highlighted that many of the side effects occur when it is taken with other medications, which she said is a concern because most patients are on other medications. There are likely to be drug interactions, she said, but “that’s not uncommon for antiepileptic medications,” and she noted that this could affect the effectiveness of the medication. The European Medical Society is also considering approval of Epidiolex and is expected to announce a decision in the first quarter of next year, according to Gover. 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. Glover said that if the results are positive, his company will apply for supplemental approval for this condition. Follow CNN Health on Facebook and Twitter See the latest news and share your comments with CNN Health on Facebook and Twitter. In the meantime, it is possible that once on the market, Epidiolex could be prescribed for conditions other than the ones it’s approved for. This is called off-label use and is a common practice with many medications. 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. Still, this approval comes as the White House is said to be reconsidering federal prohibition of marijuana and as more and more states approve it for recreational and medicinal use.