Poster Number: 3.375

Rationale: Early and effective seizure control in Dravet syndrome (DS) may be associated with improved long-term neurodevelopmental outcomes, yet data on real-world treatment patterns in the pediatric population remain limited. Stiripentol’s (STP) indication in the US was expanded from ≥2 years of age to include children down to 6 months of age in 2022, making it the only FDA approved Dravet-specific therapy in this age group. Chiron et al. demonstrated that STP initiation before 2 years of age is associated with a decrease in frequency/ duration of seizures and episodes of status epilepticus (SE)1. STP use in young children has increased, reflecting the expanded indication, evolving clinical practice, and emphasis on earlier diagnosis. This analysis describes earlier treatment approaches in patients who initiated STP at ≤3 years of age.  

Methods: A retrospective analysis was performed on US patients who initiated STP at the age of ≤3 years from August 2022 to April 2025 from a singular specialty pharmacy. Data in the analysis included age, weight, STP dosage, SKU, diagnosis code, and concomitant medications. Dosage, SKU, and diagnosis code were collected based off prescriptions dispensed to the patient, while concomitant medications were captured during discussions with patients or caregivers at initiation.  

Results: Among patients with DS who initiated STP between August 2022 - April 2025, 27% were ≤3 with an average time on therapy of 11 months. Of these, 96% were started on the sachets for oral suspension vs. the oral capsules. The average prescribed STP dose during the 1st month was 50 mg/kg/day, increasing slightly to an average prescribed maintenance dose of 51 mg/kg/day at 8 months of treatment or beyond (Table 1). At STP initiation, 77% of patients/caregivers reported use of clobazam. Thirty percent of patients had been on cannabidiol (21%) or fenfluramine (9%) prior to starting STP (Table 2). Additionally, 7% of patients reported somnolence; 4% reported weight loss/decreased appetite.  

Conclusions: This analysis represents the 1st, US-led real-world perspective of STP use in patients <3 since the label expansion in 2022.  This cohort was dosed near the recommended STP maintenance dose (50mg/kg/day) which aligns with the original RCT. Less than 10% of patients reported somnolence or decreased weight/appetite as an adverse event suggesting increased tolerability.  Nearly all patients received sachets for oral suspension, which reiterates the unmet need for a ready-to-use oral suspension. With an emphasis on early diagnosis of DS and appropriate intervention, this real-world analysis provides insights into dosing, ASM use, and formulation preferences for patients with DS started on STP ≤3 years of age. 

Funding: Biocodex, Inc.