Stiripentol Use in Dravet Syndrome Is Associated with Lower Mortality Rates in a Real-World United States (US) Cohort
Abstract number :
2.436
Submission category :
7. Anti-seizure Medications / 7E. Other
Year :
2025
Submission ID :
1348
Source :
www.aesnet.org
Presentation date :
12/7/2025 12:00:00 AM
Published date :
Authors :
Presenting Author: Carly Katterman, PharmD – Biocodex
Kelly Gwin, PharmD – Biocodex
Andrea Calvert, PharmD, BCPPS – Biocodex
Veanna Fong, N/A – Biocodex
Fabiola Garcia, N/A – Biocodex
Carla Schad, MD – Biocodex
Rationale: Dravet syndrome (DS) is a catastrophic developmental and epileptic encephalopathy characterized by recurrent generalized tonic-clonic seizures (GTCS) and a heightened risk of premature mortality. Children with DS face a 13-fold higher mortality risk compared with age-matched healthy peers, with sudden unexpected death in epilepsy (SUDEP) and status epilepticus (SE) as significant contributors, particularly in those with persistent GTCS1. Population-based studies estimate the annual mortality rate in DS to be approximately 15–16 per 1,000 patient years2. While multiple antiseizure medications (ASMs) have been approved for the treatment of DS, evidence supporting reductions in mortality is limited. In randomized controlled trials (RCTs), stiripentol (STP) demonstrated efficacy in reducing seizure burden, particularly GTCS, with nearly 40% of patients achieving seizure freedom. This analysis was conducted to better understand STP’s potential impact on mortality outcomes in a real-world US DS cohort.
Methods: Using real-world US data from patients with DS on STP therapy from August 2022 to March 2025, all-cause mortality rates were examined and compared to published epidemiologic data. Data were pulled from one exclusive distribution specialty pharmacy, PANTHERx Rare, capturing any adverse events reported as “deceased”. The number of reported deaths was analyzed relative to total patient-years of exposure to calculate the all-cause mortality rate among STP-treated patients in the US.
Results: Data were analyzed for all US patients less than 1 to 70 years of age who had received at least one dose of STP. Time on therapy ranged from 1–67 months with an average of 33 months. In this analysis a total of 19 patients with DS were reported as deceased over approximately 1,990 patient years of exposure, resulting in an estimated all-cause mortality rate of 9.55 per 1,000 patient years for STP-treated patients.
Conclusions: STP has demonstrated efficacy in reducing seizure burden in DS through combating GTCS, preventing episodes of SE, and achieving seizure freedom during RCTs. This real-world analysis suggests a lower mortality risk among STP-treated patients compared to the reported annual DS mortality rate. Given the limited data on mortality outcomes associated with DS-approved ASMs, this analysis contributes valuable insight into STP’s potential to reduce mortality risk in this high-risk population.
Funding: Biocodex, Inc.
Anti-seizure Medications