Earlier today, the Food and Drug Administration (FDA) Advisory Committee unanimously recommended the approval of the cannabis-derived drug Epidiolex. If ultimately approved, this would mark the first time the FDA greenlit a medication made with a component of psychoactive cannabis, in this case cannabidiol (CBD). Moreover, the GW Pharmaceuticals-produced drug would be the first in a new category of epilepsy-treatment options.
The FDA typically follows the recommendation of the committee, though approving the country's first cannabis-derived drug is anything but typical. That said, the unanimous decision bodes well for Epidiolex, as does the fact that the FDA awarded the drug Fast Track Designation last fall, which provided a priority review and an accelerated approval process. The deadline to complete the New Drug Application (NDA) review is June 27.
Epidiolex is designed to treat seizures associated with two rare forms of pediatric epilepsy that tend to resist traditional forms of treatment: Lennox-Gastaut syndrome and Dravet syndrome. In briefing documents released earlier this week, the FDA said it found no safety risks, adding that "the risk-benefit profile established by the data in the application appears to support approval."
In a statement, GW's Chief Executive Justin Gover said, "[The approval of Epidiolex would] provide an important treatment option for patients with LGS and Dravet syndrome, two of the most severe and treatment-resistant forms of epilepsy."
Coincidentally, the approval corresponds with the publication of new clinical studies that reaffirmed the efficacy of cannabis-based epilepsy treatment.
The Journal of Neurology, Neurosurgery, and Psychiatry published a study that examined "cannabinoids as adjunctive treatments for treatment-resistant epilepsy." Utilizing data from six controlled trials and 30 observational studies, the Australia- and England-based researchers found that CBD "was more effective than placebo at reducing seizure frequency by 50%+" and that "CBD was more effective than placebo at achieving complete seizure freedom and improving [quality of life]." In 17 of the studies, nearly half of the patients with treatment-resistant epilepsy saw their seizures cut by more than half, and 8.5 percent of the patients across eight studies stopped experiencing seizures altogether after adding CBD to their care. The median age of the patients was 16 years.
Likewise, Epilepsy Currents published the awesomely titled "Reefer to the Rescue" that examined CBD as a treatment for Dravet syndrome. The researchers found that "cannabidiol (CBD) effectively reduced seizures and autistic-like social deficits in a well-validated mouse genetic model of Dravet syndrome…. The duration and severity of thermally induced seizures and the frequency of spontaneous seizures were substantially decreased. Treatment with lower doses of CBD also improved autistic-like social interaction deficits in DS mice."
In its most recent report to the Drug Enforcement Administration, the FDA continued its support for classifying cannabis and CBD as Schedule I, a status reserved only for substances with no medical value. The approval of Epidiolex would put pressure on the agency to change the schedule status of CBD, at the very least, and hopefully for cannabis as well.