A medicine derived from the marijuana plant could become the first ever to win approval from the U.S. Food and Drug Administration. What’s more, the drug could be a major breakthrough for children who have severe forms of epilepsy.
A panel of 13 federal health advisers unanimously gave the go-ahead to Epidiolex, a drug produced by British company GW Pharmaceuticals. If the FDA takes the recommendation from the advisory panel, Epidiolex would make history as the first cannabis-based prescription drug in the U.S. with FDA approval.
Three different studies of Epidiolex show the drug reduces the number the seizures in children with Lennox-Gastaut syndrome and Dravet syndrome, two forms of childhood epilepsy that are rare and difficult to treat. Children diagnosed with either condition may have multiple epileptic seizures per day starting before age 5.
According to the Associated Press, Sam Vogelstein, a 16-year-old from California, told the FDA panel that he experienced up to 100 seizures everyday before enrolling in a trial of the experimental drug.
“I just went to South Africa for two weeks without my parents on a school trip,” Vogelstein told the panel. “I would not have been able to do that if I had not tried this medication.”
The drug is made from a chemical found in cannabis but it does not produce a high in patients, which makes it different from “medical marijuana.” In states where medical marijuana is legal, people prescribed the drug can smoke or ingest marijuana as a form of treatment. Epidiolex doesn’t contain THC, the drug in marijuana that alters the mind.
Instead, it’s a version of cannabidiol, or CBD oil, which some parents use to treat children who have epilepsy.
The president and CEO of the Epilepsy Foundation, Philip Gattone, who also testified before the FDA advisory panel, said even the chance of the drug’s approval is a sign of hope.
“Despite significant therapeutic advances in the last few years, many individuals living with epilepsy do not achieve seizure control,” Gattone said in a statement. “Epidiolex represents hope for the many individuals living with intractable seizures and rare epilepsies, who every day face incredible challenges and disabling seizures, and live with the continual risk of serious injury and death.”
The side effects of Epidiolex include diarrhea, vomiting and trouble sleeping. The most severe side effect is the potential risk of liver damage, which the FDA panel says would have to be monitored by doctors. An exact cost of the drug has not been released, but Wall Street analysts predict it could cost $25,000 a year or more.
We will know soon if the FDA approves this potentially history-making drug. The federal agency is expected to post its final decision in late June.