Phase III data for Epidiolex in tuberous sclerosis complex published in JAMA Neurology.- GW Pharma

GW Pharmaceuticals and Greenwich Biosciences announced that JAMA Neurology has published results of the company’s positive Phase III clinical trial of Epidiolex (cannabidiol) oral solution in seizures associated with tuberous sclerosis complex (TSC). Epidiolex is the first prescription, plant-derived cannabis-based medicine approved by the FDA for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or TSC in patients one year of age and older. The study found that individuals treated with Epidiolex 25 mg/kg/day or 50 mg/kg/day experienced a significantly greater reduction in TSC-associated seizures (48.6% for 25 mg/kg/day and 47.5% for 50 mg/kg/day) compared to placebo (26.5%; p<0.001 and p="0.002," respectively). this trial provided the basis for the july 2020 fda approval of epidiolex for seizures associated with tsc. the safety profile observed in the study was generally consistent with findings from previous studies of epidiolex . both doses had an acceptable safety profile, with fewer adverse events (aes) reported with 25 mg kg day than 50 mg kg day. the most common aes were diarrhea, decreased appetite, and somnolence. aes occurred in 93% of the 25 mg kg day group, 100% of the 50 mg kg day group, and 95% of the placebo group. eight patients on epidiolex 25 mg kg day, 10 on 50 mg kg day, and two on placebo discontinued treatment due to an ae. additionally, 12% of 25 mg kg day patients and 26% of 50 mg kg day patients experienced elevated liver enzymes; 79% of these patients were also taking the antiepileptic drug (aed) valproate. elevations in alt ast resolved in all patients. there were no cases meeting hy’s law criteria observed and there were no deaths in the trial.see: "add-on cannabidiol treatment for drug-resistant seizures in tuberous sclerosis complexa placebo-controlled." elizabeth a. thiele et al. jama neurol. published online december 21, 2020. doi:10.1001 jamaneurol.2020.4607>