FDA Committee Urges Approval of Cannabis-derived Epidiolex to Treat Dravet, LGS

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by Larry Luxner |

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An advisory committee of the U.S. Food and Drug Administration has recommended approval of Epidiolex, an oral cannabidiol medicine, to treat two rare forms of severe epilepsy.

The unanimous April 19 vote is non-binding, but makes it more likely that the FDA will decide on June 27 to give its blessing to Epidiolex for the treatment of seizures caused by Dravet and Lennox-Gastaut syndromes in patients ages 2 and older.

If that happens, Epidiolex would become the first-ever FDA-approved treatment for Dravet, as well as the first plant-derived cannabidiol medicine of any kind approved for prescription use in the United States. Its active ingredient, cannabidiol, is among 80 chemical compounds found in the cannabis plant, but it lacks the properties that make people high.

Mary Anne Meskis, executive director of the Dravet Syndrome Foundation, said that Epidiolex —manufactured by Britain’s GW Pharmaceuticals— was shown to reduce seizures in a recent Phase 3 clinical trial of patients with the disease, which affects roughly 1 in 16,000 births.

“Our community lacks FDA-approved treatments that have the potential to reduce seizures while minimizing side effects,” Meskis told Dravet Syndrome News in an email. “While we recognize that Epidiolex is not a cure for Dravet syndrome, we welcome it as a much-needed addition to first, second and third-line treatment options for Dravet syndrome.”

Beth Fox of Branford, Connecticut, is a parent ambassador for the organization’s northeastern region. She says the treatment is “great news” in general, though it didn’t help her 9-year-old daughter, Brenna, who has Dravet.

“Brenna was actually part of the drug trial, which started in September 2015,” Fox told us by phone. “She stayed in the trial until March 2016, when we decided to take her off Epidiolex because we didn’t see any decrease in her seizure activity, although we did see improved cognitive behavior. She was more aware and alert.”

Fox, whom Dravet Syndrome News featured in an April 3 article, said her daughter has had more than 250 unconscious seizures since she was 7 months old. Brenna has also tried and failed 14 treatments. She’s currently on four anti-seizure drugs, yet continues to have “absence seizures” every day and an average of one unpredictable and generalized seizure each week.

“When you’re trying to control your child’s seizures and they’re already taking three or four medications, you have to get rid of one to try another,” Fox told us. “Once you get to that fourth medication, it’s like you don’t want to add a fifth. The waters become too muddy and you don’t know what’s doing what.”

Justin Gover, GW’s chief executive officer, said he was “obviously pleased” by the unanimous ruling by the FDA’s independent Peripheral and Central Nervous System Drugs Advisory Committee.

“This favorable outcome marks an important milestone in our company’s unwavering commitment to address the significant unmet need for patients with LGS and Dravet syndrome, and our resolve to study Epidiolex under the highest research and manufacturing standards,” he said in a press release issued by GW’s U.S. subsidiary, Greenwich Biosciences. “We look forward to our ongoing discussions with the FDA.”

Gover later told Bloomberg that if approved, Epidiolex “will mark a sea change in the acceptability of cannabinoids as therapy.”

The ruling follows an announcement back in January that the FDA would give priority review to GW’s New Drug Application for Epidiolex. Cannabidiol, the most abundant non-psychoactive cannabinoid, has shown anti-seizure characteristics in both lab and animal models of epilepsy. It may also have antioxidant and anti-inflammatory properties.

The compound’s lack of psychoactive effects, and the preclinical evidence that it has anti-seizure properties, have generated interest in it as a potential anti-seizure therapy in humans.

Fox said she’s enthusiastic about the new therapy, even though it didn’t help her own daughter.

“What makes it significant for me is that it’s going to be an FDA-approved drug made from cannabinoid oil,” she said. “That means that instead of going through Colorado [where cannabis is legal] or a marijuana dispensary, you’re getting an FDA-approved drug you know is consistent from dose to dose, from refill to refill. A lot of families have been desperate enough to go those other routes, and they’re not necessarily getting a consistent, pharmaceutical-grade medication.”