April 23, 2018

In first, FDA panel backs marijuana-based treatment

Daily Briefing

    An FDA advisory panel on Thursday voted unanimously to recommend the agency approve an epilepsy treatment derived from cannabis.

    If approved, the treatment, called Epidiolex, would become the first marijuana-based drug approved for sale in the United States. FDA is not required to follow the panel's recommendation, though it usually does so. The agency is expected to decide whether to approve the treatment, made by GW Pharmaceuticals, by the end of June.

    About Epidiolex

    Epidiolex is a purified formulation of the cannabis plant referred to as marijuana. The formulation contains the active ingredient cannabidiol (CBD)—a chemical compound in marijuana that does not have the properties to produce the euphoric effects of the plant. As such, the formulation is designed to maximize the therapeutic properties of marijuana and minimize the so-called "high" associated with the plant.

    Epidiolex is administered as a syrup and is intended to treat severe seizures among children who have Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS)—two rare-forms of epilepsy that result in frequent and hard-to-treat seizures and put children at risk of developing intellectual or development disabilities and death, the New York Times reports.

    Clinical trial data show that patients who used Epidiolex experienced a reduction in the frequency of their seizures. In particular, the results of one clinical trial involving 120 young adults and children with DS showed Epidiolex reduced the median frequency of convulsive seizures from 12.4 per month before treatment to 5.9 per month after treatment. In comparison, individuals in the trial who received a placebo experienced minimal reductions in the frequency of their seizures, the Wall Street Journal reports.

    Research also has shown that Epidiolex is associated with some adverse events, such as gastrointestinal problems and test results that demonstrate abnormal liver function.

    FDA advisory panel backs approval

    The FDA advisory panel voted 13-0 to recommend the agency approve Epidiolex as a treatment for seizures caused by LGS and DS in patients ages two-years and older.

    According to the Washington Post's "To Your Health," the advisory panel's support for the treatment's approval came after FDA officials provided a positive review of Epidiolex. FDA in briefing documents prepared for the committee wrote, "The statistically significant and clinically meaningful results from these three studies provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with LGS and DS."

    However, FDA in the documents said it is unknown exactly how the treatment reduces seizures. FDA also reported the risk of liver injury associated with the treatment, but said such a side effect could be managed. FDA officials noted that the drug "has a negligible abuse potential."

    Comments

    Igor Grant, director of the Center for Medicinal Cannabis Research at the University of California-San Diego, said, "This is a very good development, and it basically underscores that there are medicinal properties to some of the cannabinoids." He continued, "I think there could well be other cannabinoids that are of therapeutic use, but there is just not enough research on them to say."

    Jerzy Szaflarski, a neurology professor at the University of Alabama at Birmingham who directs the university's epilepsy division, said, "It's very important that we have additional treatments because these patients have very, very difficult to control seizures."

    Christina SanInocencio, a nurse and founder of the LGS Foundation, said, "I have a brother with the disorder. I've met hundreds and hundreds of families who have kids living with it. It's so devastating. Any new medicine that comes to the market is a really big win for our community."

    Michael Privitera, a neurologist at the University of Cincinnati Medical Center who was involved in the drug's testing, said it is important for FDA to approve the treatment so parents no longer have to buy medical marijuana from dispensaries, where the quality of CBD products are not necessarily known and products might not be provided at the recommended dose (Loftus, Wall Street Journal, 4/19; McGinley, "To Your Health," Washington Post, 4/19; Kaplan, New York Times, 4/19).



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