To present the effectiveness and safety results of CBD in adult patients with TREs from the EAP.

Four-year results from the overall EAP population (mean age, 13.5 years) reported seizure reduction associated with long-term CBD use.

Patients received plant-derived highly purified CBD (Epidiolex^®; 100 mg/mL oral solution) increasing from 2–10 mg/kg/d to tolerance or maximum of 25–50 mg/kg/d. Percentage change from baseline in median monthly frequency of convulsive and total seizures and responder rates (RRs) across 12-week intervals through 144 treatment weeks were evaluated.

Within the overall safety population (n=892), 193 (22%) patients were adults (mean age [range]: 27.3 [18.0–74.5] years). Median (range) of ASMs at baseline: 3 (1–7). Most common ASMs: clobazam (37%), levetiracetam (34%), lamotrigine (33%). Median (range) CBD exposure: 733 (15–1742) days. Median (IQR) top CBD dose: 25 (21–35) mg/kg/d. In the efficacy analysis set (n=182), baseline median (IQR) monthly frequency of convulsive and total seizures was 22 (6–60) and 40 (15–100), respectively. CBD treatment was associated with median reductions from baseline of 45%–64% (convulsive seizures) and 41%–63% (total seizures). Convulsive seizure RRs (≥50%, ≥75%, and 100% reduction) were maintained through 144 weeks, ranging from 49%–64%, 27%–39%, and 7%–16% across visit intervals, respectively. Total seizure RRs showed sustained/profound reductions of ≥75% among 25%–38% of patients across visit intervals. AEs occurred in 92% of patients and serious AEs in 39%; 8% withdrew because of AEs. Two deaths occurred, both deemed unrelated to treatment. Most common treatment-related AEs (≥20%) were diarrhea (49%) and somnolence (24%). Liver-related AEs (>1%) were increased ALT, increased AST, and abnormal liver function test (5% each).