Stiripentol in Dravet syndrome: preventing prolonged seizures, reducing mortality, and improving value in care

Abstract Dravet syndrome (DS) is a rare and severe developmental and epileptic encephalopathy that begins in infancy. Although nonconvulsive seizures may occur, the syndrome is marked by frequent, prolonged convulsive seizures followed by neurodevelopmental plateau, slowing, or regression. Stiripentol (STP) is an anti-seizure medication (ASM) approved for adjunctive use in DS in countries across Europe, North America, Asia, South America, Oceania, and Africa, though it is not yet approved in most countries in Latin America. The present narrative review summarizes current evidence on the clinical efficacy, safety, and cost-effectiveness of (STP). Data from randomized controlled trials, real-world studies, and systematic literature reviews demonstrate that (STP) reduces seizure frequency and duration, increases seizure-free intervals, and helps prevent or interrupt status epilepticus (SE). These effects are particularly relevant for lowering the risk of premature mortality and improving the quality of life (QoL) for patients and caregivers. Stiripentol has also shown a consistent safety profile, including in patients treated for over 18 years. Pharmacoeconomic analyses indicate that (STP) achieves meaningful seizure reduction and seizure freedom at a lower cost compared with cannabidiol and fenfluramine, which are ASMs also approved for the treatment of DS. Cost-utility models further support (STP)'s value under commonly accepted willingness-to-pay thresholds. Taken together, current evidence supports the role of (STP) as an effective, well-tolerated, and economically favorable treatment option for individuals with DS, including from early infancy.