On Monday, the U.S. Food and Drug Administration made a surprising announcement: The agency had recommended for approval the first marijuana-based drug, called Epidiolex, to treat two types of severe epilepsy. Epidiolex is an oil containing cannabidiol (CBD)— a component of cannabis that does not cause the psychoactive high typically associated with marijuana. The drug was formally approved to treat Lennox-Gastaut syndrome and Dravet syndrome, which are particularly rare. Both cause daily seizures, are associated with developmental delays, and often don’t respond to standard anti-seizure medications. In three randomized, controlled trials, which included over 500 patients with one of the two syndromes, Epidiolex reduced seizure frequency compared to placebo when it was added on to patient’s normal treatment regimens. Like all components of marijuana, CBD is still classified as a Schedule I drug by the Drug Enforcement Administration, meaning that it’s considered by that agency to have no medical use. Possession is federally illegal, and its legal status in many states is murky. Epidiolex can’t be marketed to patients until that designation is changed, and since the FDA has signaled the compound does, in fact, have a medical use, that’s expected to happen. GW Pharmaceuticals, which developed the drug, said in a press release that it expects the compound to be rescheduled by the DEA within 90 days. It’s not clear, though, what CBD’s new classification will be changed to. FDA Commissioner Scott Gottlieb stressed in a statement from the FDA that the decision isn’t an approval of the medical use of marijuana overall. “This is the approval of one specific CBD medication for a specific use,” he said in the statement. Once the drug hits pharmacy shelves (and assuming CBD is removed from the list of Schedule I drugs), patients with Lennox-Gastaut syndrome and Dravet syndrome won’t be the only ones to benefit. Epidiolex will presumably be prescribed for other forms of epilepsy, experts say—and potentially other disorders, as well. “ If cannabidiol is moved off of Schedule I, a lot more research will be able to happen. ” — Diana Martinez, Columbia professor of psychiatry Perhaps most importantly, the anticipated rescheduling of CBD will make it easier for researchers to study its efficacy for other illnesses. Right now, though, scientists are hoping to study the effects of CBD on any number of medical conditions have to jump through dozens of hoops with the FDA, the DEA and the National Institute on Drug Abuse, which slows progress and makes it hard to build sustained research momentum. But the studies that do exist are promising: They hint that CBD has anti-inflammatory properties and helps ease anxiety. It’s also a potential therapeutic target for opioid use disorder and addition. The expected rescheduling of CBD will make it easier to iron out the questions still remaining on efficacy, side effects, and doses of the compound, Diana Martinez, a Columbia professor of psychiatry, told the Daily Beast. Martinez is in the early stages of a study testing CBD as a treatment for the pain from nerve damage caused by chemotherapy, and had to through the existing process. “It’s unfortunate, it’s really difficult,” Martinez said. “The movement with the DEA and with the FDA will make our lives easier. If cannabidiol is moved off of Schedule I, a lot more research will be able to happen.” When a new drug is approved by the FDA, the company behind it is only allowed to advertise and market their product for the specific use that the agency approved it for. However, once Epidiolex is on the market, doctors can choose to prescribe that medication off-label, for other illnesses they determine it would be able to treat. Tim Welty, professor of pharmacy practice at Drake University, said that’s probably what will happen with Epidiolex. “It’s almost certain that doctors will prescribe the drug off-label,” he told The Daily Beast. It’s hard to predict what doctors might prescribe it for, and how often. It likely won’t be the first drug that they turn to for other forms of epilepsy, Welty said. “The physicians that I know and talk to look at this as another drug to consider, not a first-line drug. It’s probably a third or fourth line drug in patients who have failed other treatments,” he said. But the pharmaceutical-grade nature of the product makes Epidiolex safer and more controlled way to take CBD. With most of the CBD products sold commercially, the concentrations of the compound the product says it has is often incorrect. According to a 2017 letter published in JAMA, around 70 percent of products sold online are incorrectly labeled. “ Even having this approved, I still think doctors will be hesitant. ” — Kevin Hill, addiction psychiatrist and director of the Division of Addiction Psychiatry at Beth Israel Deaconess Medical Center “There’s no control, and you really don’t know what you’re getting,” Welty said. Epidiolex, though, is an FDA-approved and well-regulated form of the compound, overseen and monitored in the same way as any medication. “If you have a group of people who have been interested in CBD, but have been hesitant to use the available products—now there’s an approved product,” Welty said. Epidiolex likely won’t cut significantly into the business of existing dispensaries selling marijuana-derived CBD products. Kevin Hill, addiction psychiatrist and director of the Division of Addiction Psychiatry at Beth Israel Deaconess Medical Center, told the Daily Beast that he doesn't expect people already using CBD to treat illnesses and ailments to start asking their doctors for Epidiolex en-mass. Though it hasn’t been priced yet, the drug will likely be expensive, Hill said, and he doesn’t expect that they’ll switch to a medicine that requires a doctor's sign-off. That’s because people already have cheap and easy access to CBD. It’s already used for problems like anxiety and pain in states with legal medical marijuana laws. Doctors, though, are generally cautious to prescribe or recommend the compound for the range of disorders people already use the product for. “Even having this approved, I still think doctors will be hesitant,” Hill said. That’s because, despite its popularity, there still isn’t enough evidence to support CBD’s use for anything other than epilepsy. Having a drug on the market, though, may lead to more conversations between doctors and patients about the problems and complaints that they’re interested in CBD for. “CBD may not end up being the best option, but having that conversation means that the patient is more likely to get help,” he said. Still, when choosing between drugs with evidence for a particular use and compounds like CBD, which are more speculative, the evidence-based approach should be used first. “Sometimes people exhaust their options. But we need to base initial care on clinical evidence,” Hill said. “We need to continue rigorously designed studies for other uses of CBD.” Martinez would like to see more studies on the anti-inflammatory properties of CBD, and its potential to treat diseases like rheumatoid arthritis and irritable bowel syndrome. For Hill, chronic pain and opioid use disorder are at the top of the list. And the approval of Epidiolex is a step in the right direction towards a more evidence-based approach to CBD, he said. “It lends credence to the idea that cannabinoids can be effective treatments.”<doc-sep>Medicinal benefits Getty It tastes of strawberry, but Epidiolex is the first drug approved in the US containing an ingredient from marijuana. The US Food and Drug Administration (FDA) approved the drug on 25 June for the treatment of two rare but severe forms of childhood epilepsy: Dravet syndrome and Lennox-Gastaut syndrome. Advertisement The active ingredient in the drug is cannabidiol, and it contains only a trace of the psychoactive component of cannabis, tetrahydrocannabinol, or THC. In clinical trials, Epidiolex proved effective at helping people with these conditions control their seizures. “This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies,” said Scott Gottlieb, FDA commissioner. But he warned that the organisation would continue to punish illegal marketing of cannabidiol-containing products with unproven medical claims. The UK government last week announced a review into the possible use of marijuana-based medical products, in the wake of much public debate over the use of cannabis oil to treat epilepsy. GW Pharmaceuticals, which developed Epidiolex, expects a decision on European approval early next year. In 2010, it won UK approval to sell Nabiximols for the treatment of multiple sclerosis, the first marijuana-based drug approved in the world. The company is also developing cannabis-based treatments for epilepsy, schizophrenia and glioblastoma, a form of brain cancer for which there are no reliable treatments.<doc-sep>The Food and Drug Administration approved the prescription cannabidiol medicine Monday to treat rare and severe forms of epilepsy. FDA Commissioner Dr. Scott Gottlieb also issued an immediate statement saying this wasn’t an approval of marijuana, in general — and said anyone wanting to use cannabis-based products to treat disease must prove they are safe and that they work. The new drug, Epidiolex, is a formulation of cannabidiol — CBD for short. It’s derived from cannabis but doesn’t contain the THC that recreational users value for providing “highs." “The difficult-to-control seizures that patients with Dravet syndrome and Lennox-Gastaut syndrome experience have a profound impact on these patients’ quality of life,” said the FDA’s Dr. Billy Dunn. “In addition to another important treatment option for Lennox-Gastaut patients, this first-ever approval of a drug specifically for Dravet patients will provide a significant and needed improvement in the therapeutic approach to caring for people with this condition.” The approval only covers use of the liquid drug for Lennox-Gastaut syndrome and Dravet syndrome. FDA advisers recommended the approval in April. It is not a go-ahead for other marijuana-based products, Gottlieb stressed. “This is an important medical advance,” he said. “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.” It’s very different from using pure cannabis in any form. “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,” Gottlieb said. Many companies sell cannabis-based treatments, touting them as cancer cures, pain relievers and anxiety medicines. The FDA has been warning the public against falling for these unproven claims. “The promotion and use of these unapproved products may keep some patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases,” Gottlieb said. #FDA approved the first drug with a compound derived from marijuana for treatment of seizures associated with certain rare, severe forms of epilepsy https://t.co/rgc4GNnzbZ https://t.co/ZqRbeoq5mh — Scott Gottlieb, M.D. (@SGottliebFDA) June 26, 2018 He said the FDA would review legitimate applications for approval but said the agency needed solid scientific studies that demonstrate whether a product actually works and is safe. “Such a process ensures that any new therapies from marijuana and its constituents are safe, effective and manufactured to a high and consistent quality and most importantly, that these products have been proven safe and effective for patients,” Gottlieb added. The drug has been shown to reduce seizures and return a few children to near-normal function. Dravet syndrome is a rare genetic condition that usually shows up in infants, starting with fever-related seizures and progressing to other, more severe seizure types. “Children with Dravet syndrome typically experience poor development of language and motor skills, hyperactivity and difficulty relating to others,” the FDA said. Lennox-Gastaut syndrome also causes seizures, usually starting in children aged 3 to 5. “Almost all children with Lennox-Gastaut syndrome develop learning problems and intellectual disability,” the FDA said. “Many also have delayed development of motor skills such as sitting and crawling. Most people with Lennox-Gastaut syndrome require help with usual activities of daily living.” Medical marijuana is legal in 29 states, Guam and Puerto Rico. Studies have shown that it can have some benefits in reducing anxiety, counteracting some types of nausea, and that it may relieve some symptoms of glaucoma and multiple sclerosis. The FDA has approved several marijuana-based products, including the synthetic formulation Marinol, prescribed to treat severe appetite loss and nausea in conditions such as AIDS. But marijuana products in general are subject to Schedule I restrictions by the Drug Enforcement Administration (DEA) because of the potential for abuse. Greenwich Biosciences, the U.S. subsidiary of GW Pharmaceuticals, will sell the product. "As part of the approval process, Epidiolex must be rescheduled from its current Schedule I before it can be made available to patients," the company said in a statement. "Rescheduling is expected to occur within 90 days."<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.