The Federal Drug Administration's 13-member advisory committee voted unanimously Thursday to recommend approval of the first marijuana-based medication to treat specific rare forms of epilepsy.
What are the details?
The medication, known as Epidiolex, is derived from cannabidiol, also known as CBD, and is formulated as a syrup to be taken orally. It does not contain THC, the psychoactive component of cannabis.
According to the company's website, the drug showed significant reductions in seizure activity for patients during clinical trials compared to those taking a placebo.
“The results from these three studies provide substantial evidence of the effectiveness of CBD for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome (LGS and DS),” the FDA said in briefing documents provided to the advisory committee, according to NBC News.
Lennox-Gastaut and Dravet syndromes cause uncontrolled seizures that can be life-threatening. Children with these syndromes are also at high-risk for developmental disabilities and serious injury.
“As a physician who treats LGS and Dravet syndrome, I know that patients and their families usually face significant difficulties getting seizures under control using existing therapies,” Dr. Elizabeth Thiele said in a news release. “The results from these studies suggest that this pharmaceutical formulation of cannabidiol may provide hope for a new treatment option that may be effective for some patients.”
Thiele is the director of pediatric epilepsy at Massachusetts General Hospital, professor of Neurology at Harvard Medical School, and a primary investigator for one of GW’s and Greenwich’s studies in LGS patients.
What do parents say?
Parents, whose children participated in the trials, said their child's condition and quality of life improved by taking the drug.
Polly Vanderwoude told The Washington Post that her daughter, Olivia, was a 2-month-old infant when she began having multiple seizures a day.
Olivia started Epidiolex in 2014 and now has many seizure-free days. She has improved developmentally, too. Olivia has begun smiling, making eye contact, and she has better relationships with family members, the mother said.
“It has been a lifeline,” Vanderwoude told the Post.
It's still unknown what mechanism in CBD reduces seizures, according to the FDA. GW Pharmaceuticals, a British company, would market the medication.
FDA approval could come as soon as June. Once approved, doctors could prescribe it for other conditions.