Epidiolex filed with FDA for Tuberous Sclerosis Complex.- GW Pharma

GW Pharmaceuticals announced that it has submitted a supplemental New Drug Application (sNDA) to the FDA for Epidiolex (cannabidiol) oral solution, CV. The sNDA seeks to expand the Epidiolex label to include the treatment of seizures associated with Tuberous Sclerosis Complex (TSC), a rare genetic condition. The sNDA is supported by data from a Phase III safety and efficacy study, results of which were recently presented at the American Epilepsy Society 2019 annual meeting. The study met its primary endpoint with patients treated with Epidiolex 25 mg/kg/day experiencing a significantly greater reduction from baseline in TSC-associated seizures compared to placebo (49% vs 27%; p=0.0009). Results for the 50 mg/kg/day dose group were similar, with seizure reductions of 48% from baseline vs 26.5% for placebo (p=0.0018). All key secondary endpoints were supportive of the effects on the primary endpoint. The safety profile observed was consistent with findings from previous studies, with no new safety risks identified.Comment: Epidiolex is currently approved in the U.S. to treat seizures associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome and has been granted Orphan Drug Designation from the FDA for the treatment of TSC.