British biopharmaceutical company GW Pharmaceuticals (and its San Diego-based subsidiary Greenwich Biosciences) announced today that it completed its rolling submission of a New Drug Application (NDA) for Epidiolex. The medication—a liquid formulation made from cannabis plant-derived cannabidiol (CBD)—reduces seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, two highly treatment-resistant forms of childhood-onset epilepsy.
The Food and Drug Administration (FDA) granted Rare Pediatric Disease and Orphan Drug Designations for the treatment of LGS and Dravet syndrome and a Fast Track Designation for the treatment of Dravet syndrome. Fast tracking Epidiolex provides a priority review and accelerated approval. Likewise, a rolling submission—i.e., the company completed part of the application with the rest to follow—to get the process started early rather than waiting to submit a completed application.
Data supporting the NDA includes three successful Phase 3 safety and efficacy studies, which included about 1,500 patients, many of whom took Epidiolex for more than a year. In all three studies, the patients taking the drug experienced a reduction in seizures compared to the control group that received a placebo.
"The submission of the Epidiolex NDA is an important milestone for patients with LGS and Dravet syndrome in that a potential new treatment option is within sight for these very difficult to treat and devastating conditions," said company CEO Justin Gover in a press release. "This regulatory submission is a demonstration of GW's commitment to developing innovative cannabinoid-based treatments that have the potential to address significant unmet medical needs."
GW, which expects FDA approval, plans to roll out Epidiolex in the U.S. next year.
The company is also testing other Epidiolex applications, including treatment for tuberous sclerosis complex, which is currently in Phase 3 trials. Another GW product, Sativex—a tetrahydrocannabinol (THC) and CBD oral spray that treats muscle stiffness and spasms caused by multiple sclerosis (MS)—already has regulatory approval in 30 countries, including France, Germany, Australia and Canada, but not the United States. Other GW medications in the pipeline target a wide range of disorders such as autism, glioma and schizophrenia, among others.
What does this mean for cannabis? For starters, Epidiolex would become the first ever FDA-approved treatment for Dravet syndrome. Second, it would put more pressure on the government—and the FDA in particular—to reschedule cannabis.
Both the plant itself and CBD extracts are currently Schedule I substances, which means they are fully prohibited and deemed to have no medical value. In 2016, the FDA recommended that the DEA should not reschedule cannabis, but the approval of Epidiolex would provide evidence of medical value for CBD, if not the whole plant. Since doctors cannot prescribe Schedule I substances, some type of regulatory change must occur to make Epidiolex available to patients. To what extent the change affects other cannabis products is to be determined.