Most patients with those diseases have uncontrolled daily seizures despite treatment, putting them at high risk for developmental disabilities, serious injury and premature death, the FDA staff said in background materials released this week.
The unanimous support of the 13-member FDA advisory committee seemed assured after agency staff members gave the drug a positive review, saying clinical trials showed it was effective at reducing seizures, and side effects, including possible liver complications, could be managed. The precise mechanism by which CBD reduces seizures isn't known, the FDA said.
The drug does not contain THC, the psychoactive component in cannabis. The FDA staff said the drug “has a negligible abuse potential.”
The FDA isn't required to follow the recommendation of its outside advisers, but it usually does. The agency has indicated that it would act by late June on the application from GW Pharmaceuticals, a British company that would market the drug.
An FDA approval would be targeted to specific epilepsy patients, but once approved, the drug could be prescribed for other uses. And it could pave the way for other cannabis-based drugs, analysts say.
Several parents whose children have been participating in clinical trials of the drug said that their children's lives have been transformed by the medication.
Polly Vanderwoude said her daughter, Olivia, began having seizures in 2010 when she was 2 months old, and had multiple seizures a day. But since Olivia began taking Epidiolex in 2014, her mother said, there have been “great improvements,” including many days when she did not have any seizures at all. In addition, her daughter now is smiling and making eye contact and has closer relationships with family members, Vanderwoude said.
“It has been a lifeline,” Vanderwoude said.
Michael Privitera, a neurologist at the University of Cincinnati Medical Center who was involved in the testing of the drug, said it was a major advance, and it was important that the drug be approved so parents don't have to buy CBD from medical marijuana dispensaries, which don't necessarily know the quality of CBD products or the recommended dose.