USL255 is a once-daily, extended-release formulation of the well-established antiepileptic drug topiramate that was recently approved by the US FDA. As a capsule formulation, USL255 can be swallowed intact or opened and sprinkled onto soft food for patients with swallowing difficulties, including children (≥2 years old) and older patients. USL255 has been evaluated in seven key Phase I and III studies. Compared with immediate-release topiramate taken twice daily, once-daily USL255 provides equivalent topiramate exposure with a 26% reduction in plasma fluctuations. A multinational, Phase III, randomized, double-blind, placebo-controlled clinical trial in patients with refractory partial-onset seizures (PREVAIL) demonstrated that USL255 (200 mg/day) significantly improved seizure control and clinical outcomes versus placebo. USL255 is generally safe and well-tolerated, with a low incidence of neuropsychiatric and neurocognitive adverse events. These data suggest that USL255 may provide a useful treatment option for seizure control with convenient once-daily dosing.
Keywords: Lennox–Gastaut syndrome; antiepileptic drug; extended-release; partial-onset seizure; pediatric; primary generalized tonic-clonic.