Update 6/25/18: After unanimous approval from an expert panel in April, the FDA approved Epidiolex. The headline has been updated to reflect this news. FDA Commissioner Scott Gottlieb noted in a statement that “that this is not an approval of marijuana or all of its components” and said the agency remains “concerned about the proliferation and illegal marketing of unapproved CBD-containing products with unproven medical claims.
Today, an expert panel of advisors to the Food and Drug Administration recommendedthat the agency approve a marijuana-derived medicine used as a treatment for severe seizures in children with epilepsy. Pending a final decision by the Food and Drug Administration, expected by late June, the drug could be the first marijuana-based medicine to receive government approval in the United States. The FDA is not bound to follow the recommendations of its outside experts, but it usually does.
The agency’s 13-member health panelunanimously recommended the medication based on three studies showing its effectiveness in treating two rare forms of childhood epilepsy; by voting in favor of the drug, they agreed that its benefits outweighed the risks. Called Epidiolex, it’s produced by a British company, GW Pharmaceuticals, and comes in a strawberry-flavored liquid form similar to the cannabidiol (or CBD) used to treat a variety of ailments. Some parents have already been using CBD oil to treat their children with epilepsy, though it’s obviously a big deal to have that treatment approved and regulated by the FDA.
It’s also worth noting that CBD doesn’t produce a “high”—it’s chemically different from the mind-altering THC (tetrahydrocannabinol) found in marijuana. Patients treated with the drug won’t feel any weed-like effects. The product is derived from the company’s proprietary strain of marijuana that’s been bred to have high CBD content.
The panel recommended approval based in part on clinical trials suggesting Epidiolex is an effective treatment for Dravet syndrome, a severe form of epilepsy stemming from a rare genetic mutation. It’s marked by frequent and prolonged seizures within a child’s first year, and some 20 percent of those afflicted die. Several high-profile cases showed the potential of CBD oil to treat the condition, and more formal research followed, eventually leading to today’s decision. (Patients with Lennox-Gastaut, another severe form of epilepsy, have also responded well to Epidiolex treatment; the panel was voting on whether to recommend approval for these two conditions.)
CBD has long occupied a legal gray area, though. It’s easy to buy online (though you don’t always get what you pay for), with manufacturers saying their products are derived from hemp, which, unlike marijuana, is legal to cultivate. People use it for pain relief and even as a sunburn salve, though the FDA recently called out a number of companies for claiming their CBD products could treat or cure cancer.
The panel did note that Epidiolex comes with a potential risk of liver damage, but prescribing doctors could monitor patients accordingly. More commonly, the drug might cause diarrhea, vomiting, fatigue, and sleep problems.
While more than two dozen states recognize medical marijuana, the FDA has not approved any medicinal use for the plant at the federal level. The agency has approved synthetic versions of THC and CBD for treating side effects of chemotherapy, though under Drug Enforcement Agency rules THC is classified as a Schedule II drug, meaning its use is highly restricted. Marijuana is currently Schedule I, the most restrictive classification for drugs, considered by the DEA to have “no currently accepted medical use and a high potential for abuse.” According to Bloomberg, FDA staff indicated at the meeting that if Epidiolex is ultimately approved for sale, CBD would have to be rescheduled.
How much this will cost patients isn’t yet clear, though Wall Street analysts say Epidiolex could run more than $25,000 per year.
It seems unlikely that an approval will open a large market for government-sanctioned, marijuana-based medicine. With the FDA’s approval, doctors could prescribe Epidiolex to the small number of people with two rare forms of epilepsy—though they could also prescribe it for “off-label” treatment of other conditions. It may also spur more CBD research from deep-pocketed pharmaceutical companies who recognize an opportunity.