U.S. health regulators have approved the first prescription drug made from marijuana, a milestone that could spur more research into a drug that remains illegal under federal law.
The Food and Drug Administration approved the medication from GW Pharmaceuticals on Monday, to treat two rare and serious forms of childhood epilepsy: Lennox-Gastaut syndrome and Dravet syndrome, that begin in childhood but can persist in adulthood. The strawberry-flavored syrup is a purified form of a chemical in the cannabis plant — but not the one that leads to the high. It is not yet clear why the ingredient, known as CBD, reduces seizures in some people.
The British drugmaker studied the drug in more than 500 patients with hard-to-treat seizures, overcoming numerous legal hurdles to conducting research with cannabis. FDA officials have stated that the drug reduced seizures when combined with older epilepsy drugs.
The drug is made from purified cannabidiol, or CBD, a compound found in the cannabis plant. The drug will be marketed under the brand name Epidiolex. CBD has medicinal effects, but it does not cause the mind-altering high that comes from THC, the primary psychoactive component of marijuana.
The FDA says this is the first drug approved in the U.S. that contains a purified substance derived from marijuana. The agency has previously approved drugs made from synthetic versions of THC and other marijuana constituents. “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 FDA Commissioner Scott Gottlieb during a call with reporters about the approval.
Several states have legalized CBD oil specifically for the treatment of intractable epilepsy or seizure disorders. As media outlets have previously reported, CBD supplements are available widely online and in dispensaries in the form of oils or tinctures, and CBD oil has gained popularity with consumers as a remedy for a variety of other ailments. However, the legal status of these products is uncertain, as is their quality; they are not regulated the way pharmaceutical drugs are, so the consistency and dose can vary widely.
Having an FDA-approved, pharmaceutical-grade CBD drug will open up a new treatment option for epilepsy patients by delivering a high-quality, consistent dose of CBD, says Robert Carson a pediatric neurologist at Vanderbilt University who treats patients with epilepsy. “Our biggest concerns with the artisanal [or supplement] versions of CBD were related to the consistency,” Carson says. “We can’t guarantee the consistency.” Carson says he will likely prescribe Epidiolex going forward. “I’m always excited about the potential for a new therapy that has been well-studied and has a great potential for benefit,” he says.
Several researchers are studying the potential of CBD to treat psychiatric conditions. A clinical trial is underway to test whether CBD can be an effective treatment for people with post-traumatic stress disorder and alcohol use disorder, and another trial will determine whether CBD could help prevent relapse in opioid abusers.
The approval of Epidiolex may help open the door to more CBD research, as it helps to lift one regulatory hurdle. Until now, the Drug Enforcement Administration has classified CBD as a Schedule 1 substance. Like other drugs in this category, which include heroin and LSD, these drugs are considered to have no medical use and a high potential for abuse.
But now, with the approval of a CBD drug, the DEA will change this, according to Dr. Douglas Throckmorton, deputy director of regulatory programs at the FDA’s Center for Drug Evaluation and Research, “The DEA will need to make a different scheduling decision for CBD…because it now has an accepted medical use,” he said during a conference call with reporters. He said the reclassification is underway now.