The Food and Drug Administration (FDA) on Monday gave a green light to the country's first drug derived from marijuana. Epidiolex, manufactured by GW Pharmaceuticals, is intended to treat seizures associated with two rare and severe forms of epilepsy that begin in childhood. The drug is made of cannabidiol (CBD), a component of marijuana that doesn’t give users a high. The approval could spur more research into marijuana products, though marijuana itself remains illegal. ADVERTISEMENT “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,” FDA Commissioner Scott Gottlieb said in a statement. In a separate statement, Gottlieb stressed the importance of using proper research on the medical uses of marijuana and noted the approval doesn't mean the agency will be lowering its approval standards for marijuana. "This is an important medical advance. But it’s also important to note that this is not an approval of marijuana or all of its components," he said. "This is the approval of one specific CBD medication for a specific use." Medical marijuana is available in about half of states. But federal regulations classify CBD as a Schedule 1 drug, which means it has no medical value and a high potential for abuse, because it is a chemical component of the cannabis plant. This means the Drug Enforcement Administration (DEA) will have to reclassify it before GW can begin marketing Epidiolex. The FDA said it is providing medical and scientific information and recommendations to the DEA about CBD. Updated at 1:46 p.m.<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. Gover said the approval signals “validation of the science of cannabinoid medication.”<doc-sep>(Reuters) - The U.S. health regulator approved GW Pharmaceuticals Plc’s epilepsy treatment on Monday, making it the first cannabis-based drug to win approval in the country and opening floodgates for more research into the medicinal properties of cannabis. FILE PHOTO: A flowering marijuana plant. REUTERS/Chris Wattie The drug’s approval permits its use in patients aged two years and older with Dravet Syndrome (DS) and Lennox-Gastaut Syndrome (LGS), rare childhood-onset forms of epilepsy that are among the most resistant to treatment. “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 Food and Drug Administration Commissioner Scott Gottlieb. The drug, Epidiolex, is made up of cannabidiol (CBD), one of the hundreds of molecules found in the marijuana plant, and contains less than 0.1 percent of tetrahydrocannabinol (THC), the psychoactive component that makes people high. GW Pharma grows its own supply of cannabis in specialized glass houses in the United Kingdom to ensure uniformity in the genetic composition of the plants, which are then processed into a liquid solution of CBD. Although THC can induce paranoia, anxiety and hallucinations, CBD has the opposite effect and has been cited by scientists as a potential treatment for mental health issues. While supporters of legalizing marijuana say the decision is a step in the right direction, businesses reliant on the plant must contend with the federal government’s ban on its use. Based on the potential for abuse, the Drug Enforcement Administration (DEA) categorizes chemicals into five schedules, with Schedule 1 substances - like marijuana and heroin - considered the most deadly and deemed to have no medical benefits. As a result, Epidiolex’s launch remains at the discretion of the DEA, which must now evaluate the drug and consider reclassifying it as a substance that has medical properties, so as to allow GW to begin selling it. GW said it expects the reclassification to occur within 90 days. The company has not yet set a price for the drug and said it would work with insurance providers to ensure the medicine would be covered under health plans. GW’s Nasdaq-listed shares marginally fell in afternoon trading. The stock has run up nearly 50 percent in the past 12 months. Most patients with LGS and DS require multiple seizure medications and the majority are resistant to currently approved anti-epileptic drugs. The two epilepsy forms are severe and associated with high rates of mortality. Some LGS patients have to wear helmets to avoid brain injuries from “drop seizures”, where muscles suddenly become limp and cause standing patients to collapse. Epidiolex would also be the first approved therapy for DS, treatments for which are currently limited to a combination of seizure medication and drugs to prevent emergencies. Treatments available for both disorders are far from perfect and some patients resort to buying “self-prescribed” CBD online or from unregulated vendor sites, Dr. Pavel Klein, founder of the Mid-Atlantic Epilepsy and Sleep Center, said. “I hope patients have conversations with their physicians about whether this product (Epidiolex) provides them with treatment they have been looking for with other unapproved products,” said Douglas Throckmorton, deputy director of regulatory programs at the FDA’s Center for Drug Evaluation and Research. (This version of the story has been refiled to correct paragraph 12 to show DEA must make a decision within 90 days of FDA approving a medicine, instead of there is no time limit.)<doc-sep>
Image : AP The US Food and Drug Administration has approved the use of Epidiolex to treat two severe forms of epilepsy, making it the first cannabis-based drug to be legalized in the United States. It’s welcome news—but patients won’t have access to the medicine until the Drug Enforcement Agency changes the Schedule 1 designation of cannabis. Epidiolex, an oral drug developed by GW Research Ltd., now has FDA approval to treat two rare, severe forms of epilepsy—Lennox-Gastaut syndrome and Dravet syndrome—in patients over the age of two. Back in April, an FDA panel unanimously recommended that Epidiolex be approved for medical use, so this decision is not altogether unexpected. In addition to being the first cannabis-based drug to be approved in the US, Epidiolex is now the first FDA-approved drug to treat Dravet syndrome. “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. And, the FDA is committed to this kind of careful scientific research and drug development,” said FDA Commissioner Scott Gottlieb in a statement. “Controlled clinical trials testing the safety and efficacy of a drug, along with careful review through the FDA’s drug approval process, is the most appropriate way to bring marijuana-derived treatments to patients.” This syrup-like medicine contains an active ingredient from weed called CBD, or cannabidiol. CBD is not the chemical component in cannabis that gets a person high (that distinction goes to tetrahydrocannabinol, or THC), so Epidiolex has no psychoactive properties. That said, CBD is still considered a Schedule I substance in the United States under the Controlled Substances Act because it comes from the marijuana plant. According to the DEA, Schedule I drugs are substances or chemicals defined as “drugs with no currently accepted medical use and a high potential for abuse,” a classification that includes such drugs as heroin, LSD, and ecstasy. This means that, despite the FDA’s go-ahead, Epidiolex won’t be available to patients until CBD is rescheduled by the DEA, which is expected to happen within 90 days, according to GW Research Ltd. CBD has been credited with an array of medical benefits, from treating autism and anxiety to depression and cancer, but many of these claims remain unsupported by evidence. Recent data, however, suggests it’s effective for treating seizures, including Dravet syndrome and Lennox-Gastaut syndrome. In the case of Epidiolex, the drug’s efficacy was studied in three clinical trials involving 516 patients. When taken with other medications, it reduced the frequency of seizures compared to placebos. Dravet syndrome is a rare genetic condition that appears in young children, often during the first year of life. It’s associated with fever-related seizures, poor language development and motor skills, hyperactivity, and trouble relating to others. It has a high mortality rate, with patients sometimes dying before the age of 10. Lennox-Gastaut syndrome, which also starts in early childhood, is associated with tonic seizures, where muscles contract uncontrollably. It’s also linked with learning problems, intellectual disabilities, and compromised motor skills. “For those living with intractable seizures caused by LGS and Dravet syndrome, Epidiolex represents a true medical advancement,” said Philip Gattone, president and CEO of the Epilepsy Foundation, in a statement. “Clinical development for these rare and severe conditions is essential, and today’s news brings hope for these patients and their families that a new treatment option may have the potential to help better control their seizures.” Unfortunately, Epidiolex does have unpleasant side-effects, including sleepiness, lethargy, decreased appetite, diarrhea, rashes, sleep problems, and other health issues. “As is true for all drugs that treat epilepsy, the most serious risks include thoughts about suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression and panic attacks,” writes the FDA. “Epidiolex also caused liver injury, generally mild, but raising the possibility of rare, but more severe injury. More severe liver injury can cause nausea, vomiting, abdominal pain, fatigue, anorexia, jaundice and/or dark urine.” Accordingly, the FDA has ruled that Epidiolex must be administered with a patient Medication Guide that describes the drug’s various uses and risks. The side-effects produced by Epidiolex weren’t bad or frequent enough to prevent FDA approval, and the impacts of side-effects can be mitigated through the careful monitoring of symptoms. The pall that has surrounded cannabis for so many years is finally being stripped away.