Epidyolex

Epidyolex is indicated for patients aged 2 years and older. Clinical trials for Epidyolex included 715 randomized patients across 23 sites, with 396 patients diagnosed with Lennox-Gastaut Syndrome (LGS). LGS accounts for 1-4% of childhood epilepsies, with 90% of cases being drug-resistant and over 75% experiencing cognitive impairment by 5 years post-onset. All-cause mortality in LGS is 14 times greater than in the general population.

In efficacy trials, Epidyolex demonstrated a median percentage change from baseline in total convulsive seizure frequency of -38.94%, compared to -13.29% for placebo. The estimated median difference in percentage change was -22.79, with a p-value of 0.0123. For drop seizures, the 10 mg/kg/day dose of GWP42003-P showed a statistical significance of 0.0016 in reduction versus placebo, while the 20 mg/kg/day dose showed a statistical significance of 0.0047. At baseline, the median drop seizure frequency for the 10 mg/kg/day GWP42003-P group was 86.90 per 28 days, for the 20 mg/kg/day GWP42003-P group was 85.53 per 28 days, and for placebo was 80.25 per 28 days.

Safety findings included 3 patients in the CBD-OS group experiencing emergent status epilepticus. Inpatient hospitalizations due to epilepsy occurred in 8.2% of patients in the CBD-OS group, compared to 1.7% in the placebo group. A total of 13 patients withdrew from the trial, with 9 withdrawals from the 20 mg/kg/day CBD-OS group, 2 from the 10 mg/kg/day CBD-OS group, and 2 from the placebo group. Withdrawals due to adverse events were 4 in the 20 mg/kg/day CBD-OS group, 1 in the 10 mg/kg/day CBD-OS group, and 1 in the placebo group.