FDA on Monday for the first time approved a drug derived from marijuana.
About the drug
The drug, called Epidiolex and manufactured by GW Pharmaceuticals, was approved to treat seizures in patients two and older who have Dravet Syndrome (DS) or Lennox-Gastaut Syndrome (LGS), which are rare and severe forms of epilepsy that begin in childhood. Patients with DS and LGS often take multiple types of seizure medication and are resistant to the drugs. According to Reuters, Epidiolex is the first FDA-approved drug to treat DS.
The drug is made of cannabidiol (CBD), a component of marijuana that does not give users a high. According to FDA, GW tested Epidiolex's effectiveness in three randomized, double-blind, placebo-controlled clinical trials involving 516 patients.
FDA said Epidiolex must be dispensed with a patient Medication Guide that outlines important risk factors and other information. For instance, FDA said the new drug's potential risks include depression, increased suicidal thoughts, feelings of agitation, and panic attacks—side effects that are shared by other epilepsy medications. FDA said the trial also suggests the drug carries an increased risk of liver injury.
The company has not yet set a price for Epidiolex and said it would work with insurers to make sure it is covered by most health plans.
However, before GW can sell the drug in the United States, the Drug Enforcement Administration (DEA) will need to evaluate it and determine whether the agency will reclassify cannabidiol, which remains a Schedule I drug under federal law. Schedule I drugs are considered to have a high risk of misuse and no medicinal benefits. According to Reuters, GW expects DEA will reclassify the drug within 90 days.
FDA Commissioner Scott Gottlieb in a statement called the approval "an important medical advance." He said, "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."
However, he cautioned that the approval "is not an approval of marijuana or all of its components. This is the approval of one specific CBD medication for a specific use" (Joseph, STAT News, 6/25; Weixel, The Hill, 6/25; Mathias/Mishra, Reuters, 6/25).
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