To assess real-world effectiveness, safety and tolerability of perampanel (PER) when used as early add-on therapy in everyday clinical practice.

Real-world clinical practice data complement evidence from clinical trials. PER is indicated in the US for treatment of focal-onset seizures (patients aged ≥4 years) and as adjunctive therapy for generalized-onset tonic-clonic seizures (patients aged ≥12 years).

Patients treated with PER as early add-on therapy for focal and/or generalized seizures were identified from interim pooled analysis of data from 18 clinical practice studies/work groups. Retention was assessed after 3, 6 and 12 months. Effectiveness assessments included seizure freedom rate (no seizures since at least prior visit) and responder rate (≥50% seizure frequency reduction), assessed at last visit by seizure type (focal or generalized). Safety and tolerability were assessed by evaluating adverse events (AEs).  

A total of 430 patients treated with PER as early add-on therapy for focal and/or generalized seizures were identified (52.1% male; mean age, 41.9 years; mean epilepsy duration, 12.2 years). Seizure types at baseline were focal only (85.0%), generalized only (14.3%), and focal and generalized (0.7%). Patients received median of 1 concomitant antiepileptic drug at baseline and at last visit. At 3, 6 and 12 months, retention rates were 94.4%, 86.1% and 79.3%, respectively. Mean time under PER treatment was 12.0 months. At last visit, seizure freedom rates in patients with focal and generalized seizures were 34.8% and 56.1%, respectively, and corresponding responder rates were 80.2% and 80.7%, respectively. AEs were reported for 40.9% of patients; most frequently reported AEs were behavioral AEs (aggression/anger/irritability; 15.1%), somnolence (12.9%) and dizziness/vertigo (10.6%). Overall, 13.4% of patients discontinued due to AEs. 

PER was effective and generally well tolerated as early add-on therapy in patients with focal and/or generalized seizures treated in clinical practice.