Adjunctive Epidiolex Shows Real-World Effectiveness for Focal Epilepsy

According to real-world results from an expanded access program (EAP) study population presented at the American Epilepsy Society (AES) 2024 Annual Meeting, adjunctive therapy with Epidiolex (cannabidiol; Jazz Pharmaceuticals, Dublin, Ireland) was effective for treatment-resistant focal epilepsy. Participants treated with Epidiolex showed significant reductions in focal and total seizures through 144 weeks, with 93% of participants reporting adverse events (AEs) at any point during 240 weeks of follow-up.

In the EAP, initiated in 2014 across 35 US sites, patients with treatment-resistant epilepsy received adjunctive therapy with Epidiolex as a 100 mg/mL oral solution at a starting dose ranging from 2 mg/kg/d to 10 mg/kg/d, which was increased up to tolerance or a maximum dose range of 25 mg/kg/d to 50 mg/kg/d, depending on site and institutional review board approval. There were 150 participants from the EAP who were identified as having confirmed focal etiology or a focal epilepsy diagnosis who were included in this analysis. Researchers assessed percent change in median monthly seizure frequency from baseline for focal and total seizures through 144 weeks.

In terms of results from this efficacy analysis set:

• Median monthly seizure frequency at baseline was 27 for focal seizures and 56 for total seizures.
• Focal seizure frequency was reduced by a median of 54% to 77%.
• Total seizure frequency was reduced by a median of 54% to 75%.
• 51% to 72% of participants with focal seizures reported a reduction of ≥50% in seizure frequency.
• For total seizures, 50% to 64% of participants achieved a reduction of ≥50% in seizure frequency.
• 93% of patients reported AEs at any point during the 240-week safety period.
• 38% of patients reported serious AEs, and 8% of patients discontinued Epidiolex due to AEs.
• Diarrhea (47%), convulsion (25%), and somnolence (21%) were reported in ≥20% of patients.
• The most common antiseizure medications (ASMs) used by patients in the EAP were clobazam, levetiracetam, and lamotrigine.