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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.

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“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.

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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.

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“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. This latest development is good news, but it’s sad to think that CBD-based drugs, and likely many other medical uses of the plant, were unnecessarily delayed for so long.

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[FDA, Greenwich Biosciences]