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This Could Be the First Cannabis-Derived Drug to Win FDA Approval

Epidiolex shows great promise in relieving two severe forms of epilepsy.

A girl with Dravet syndrome receives treatmentFrank Gunn/ZUMA Press/NewscomEpidiolex, a cannabidiol drug used to treat rare, severe forms of epilepsy in children, has moved one step closer to approval by the Food and Drug Administration, which could happen as soon as June. If approved, Epidiolex will be the first cannabis-derived drug recognized as a medicine under federal law. The FDA's announcement comes days before an independent, expert panel will vote on its safety.

Because of Epidiolex's success in treating diseases with no alternative treatments, the FDA gave it "priority review" status in December, meaning the agency will review the application for the drug within six months (compared to 10 months under standard review). After the advisory board makes a recommendation to the FDA, the agency has until June 27 to reach a decision.

The FDA staff report to the advisory committee says Epidiolex reduced seizures in a "clinically meaningful and statistically significant" way for patients suffering from Lennox-Gastaut syndrome and Dravet syndrome. The results of those studies provide "substantial evidence" of the drug's efficacy, the report says.

Both Dravet Syndrome and Lennox-Gastaut are difficult to treat, as patients experience several types of seizures and are often resistant to medication. Patients who participated in the studies had taken other medications without adequate relief.

Over two weeks, total seizures among Lennox-Gastaut patients who took Epidolex fell, on average, by 38 percent in one study and 44 percent in the other, while placebo patients experienced a drop of 18.5 percent and 23.5 percent, respectively. About 40 percent of patients in the cannabidiol (CBD) treatment groups saw a 50 percent or greater reduction in "drop seizures"—violent seizures that cause the upper body or full body to go limp, resulting in falls or injuries. Injuries from falling and poorly controlled seizures are cited as risk factors in the premature deaths of Lennox-Gastaut patients.

The author of one Lennox-Gastaut study, Elizabeth Thiele, director of pediatric epilepsy at Massachusetts General Hospital, called Epidolex "life changing" in an interview with The Washington Post. "One child who comes to mind had multiple seizures a day," Thiele said. "She had been on every medication possible. She is now talking about college options. She would have never had that conversation before."

In a Dravet syndrome study, cannabidiol cut the median number of monthly convulsive seizures in half, from 12.4 to 5.9. The median number of seizures in the control group fell only slightly, from of 14.9 to 14.1 per month.

People with Dravet syndrome require constant care because of their frequent seizures, and 15 to 20 percent of them do not reach adulthood, dying at an average age of 8. The FDA has not approved any treatments specifically for Dravet syndrome. Since Epidiolex, made by the British company GW Pharmaceuticals, is not available yet in the United States, many families with children who suffer from Dravet travel to states where they can legally obtain CBD oil.

Although 29 states and the District of Columbia allow medical use of cannabis, the federal government still classifies it as a Schedule I drug, meaning it is not legal for any use. A synthetic version of THC, another compound found in marijuana, has been available by prescription since 1985. But Epidiolex would be the first FDA-approved medicine derived directly from cannabis.

Currently 1,100 epilepsy patients obtain Epidiolex through the FDA's Investigational New Drug program, which lets patients try medications that have not yet received FDA approval. If the FDA approves Epidolex, it would increase medical access and offer families an alternative to CBD oil, which is legal only in some states. It would also spur further research on cannabis-based medicine.

"The important thing for us is that patients like this deserve a pharmaceutical solution," GW Pharmaceuticals Chief Executive Justin Glover told the Post. "They should not be moving across the country. They deserve the right to have access."

Photo Credit: Frank Gunn/ZUMA Press/Newscom

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  • Fist of Etiquette||

    Today New Jersey Democrat Chris Christie is weeping.

  • Unicorn Abattoir||

    cannabidiol cut the median number of monthly convulsive seizures in half, from 12.4 to 5.9.

    Given the choice between this and keeping MJ illegal, he'd still be against legalizing it.

  • DajjaI||

    This is horrible. These kids can get addicted to these drugs and are likely to resort to crime to obtain them. Also parents, family members and neighbors may be unable to resist the drug's lure and get addicted too!

  • Tom Bombadil||

    OBL could take some satire lessons here.

    In the debate, who get's to yell "But what about the children"?

  • Citizen X - #6||

    Both sides do. Then, shortly after the shouting reaches a crescendo, they fuck.

  • Josh Melton||

    I hope you're trolling, because otherwise you're just ignorant. CBD isn't THC. Go read before you make any more comments.

  • Juice||

    Damn, that's an effective placebo.

  • Don't look at me.||

    This isn't possible. Marijuana is a schedule 1 dangerous and powerful gateway drug with no medical uses. My government has told me so and has jailed millions of fools that dare to even touch this evil weed. It was for their own protection lest they become slobbering addicts to the devil weed. Well I'm sure they had good intentions anyway.

  • Rich||

    "They deserve the right to have access."

    "Deserve"? "Right"? "Access"?

    Talk about word salad!

  • Citizen X - #6||

    Seriously. "Deserve" implies something granted, rather than something inalienable. The right to put whatever you want into your own body - i.e. the right to own yourself - comes from the fact of existence, not from the whim of a ruler.

  • gormadoc||

    He's talking about legal rights, here. Currently they do not have the legal right, though they should.

  • Shirley Knott||

    "Legal right" is an oxymoron.
    Either you have the right or you don't, regardless of the law.
    Either you have permission from the slavers or you don't, regardless of your rights.

  • leninsmummy||

    thank you.

  • Scarecrow Repair & Chippering||

    "Multiple seizures a day" -- could this explain Chuckie's turnabout?

  • Citizen X - #6||

    Cannabidiol supposedly also shrinks some tumors. If Chuck Schumer's cup size has noticeably decreased in recent months, that might be your answer.

  • Hank Phillips||

    But... what about all the research and investment that went into drugs intended to replace cannabis and thereby please regulators, prohibitionists, lobbyists, the Wizened Christian Temperance Union and Harry Anslinger? Isn't that how Thalidomide was developed?

  • Hank Phillips||

    David Carradine's satire on Dopenhagen would make good alt-text for the photo: Happy Daze!

  • silver.||

    So, I think the only difference here between this drug and OTC CBD oil is FDA approval. In states where CBD is prohibited, will the pharmaceutical version be commercially available without having to change the laws? Does FDA approval override state-level prohibition?

    See. This is why we should just not prohibit things in the first place. Too many laws.

    An American should be able to go to any state with an AR, a giant vibrator, and a kilo of yeyo in their duffle without having to consult 6 lawyers.

    Is that so much to ask?

  • Citizen X - #6||

    Alcohol, Tobacco, Firearms, and Explosives should be a convenience store chain, not a government agency.

  • Citizen X - #6||

    It's maybe appropriate that CBD oil is one thing that i've found that keeps me from flipping all of my shit at how goddamn fucking stupid this dumbassery all is.

  • Josh Melton||

    I support you.

  • target||

    Since is Epidiolex is cannabis-derived, and cannabis is listed as schedule 1, doesn't this mean that it can't be legally sold even with FDA approval until cannabis is rescheduled?

  • Josh Melton||

    By the DEA's very definition: "Schedule I drugs, substances, or chemicals are defined as drugs with no currently accepted medical use and a high potential for abuse," cannabis would have to be dropped because we would have an FDA-approved medical use. Wouldn't that be awesome!?!

  • Josh Melton||

    Such a wonderful move in the right direction for those suffering. I hope choices are made in the interest of those who need CBD to control their seizures, and that this immediately drops cannabis off schedule 1 (see the DEA definition), restoring states rights on this particular topic.

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