Abstracts

Rationale: Dravet syndrome (DS) is a rare, drug-resistant epilepsy that typically presents within the first year of life. Dravet syndrome manifests in healthy infants and is often linked to SCN1A mutations that result in multiple seizure types and increased mortality risk. Outside of seizure burden, patients can experience motor, cognitive, intellectual, and behavioral deficits. Stiripentol is approved in the US for Dravet patients 6 months or older (weighing 15 lb. or more) taking clobazam. Information regarding dosing of stiripentol with Dravet-specific medications, including fenfluramine and cannabidiol, with trends in age, formulation, and diagnosis has been limited. To better understand the holistic approach to stiripentol's utilization, an analysis focusing on these differences was performed.

Methods: A retrospective analysis was performed on all new US patients who were prescribed stiripentol from August 2022 to April 2024. Data in the analysis included age, weight, stiripentol dosage, SKU, diagnosis code, and concomitant medications. Medication, diagnosis, and demographic data were collected based off the last prescription dispensed to the patient. Concomitant medications were captured during discussions with patients or caregivers at initiation and during monthly pharmacy follow-ups.

Results: Dosing data was analyzed in all US patients ages less than 1 to 53 years old receiving at least one dose of stiripentol. The overall average dose was 38.16 mg/kg/day, further broken down by age group and months on therapy in Table 1 for DS patients. 39.52% of DS patients were taking Diacomit 250mg packets, while 43.58% of non-DS patients were utilizing Diacomit 250mg capsules in their dosing regimens. Concomitant use of clobazam, cannabidiol, and fenfluramine with stiripentol at both the initiation and refill call is highlighted in Table 2.

Conclusions: This analysis of prescription data provides several new insights. Most prescription doses were lower than the approved dosing for stiripentol of 50 mg/kg/day, with higher doses typically in younger patients. It was reported that 50% of patients who are currently taking stiripentol are not taking concomitant clobazam. This analysis shows the percentage of patients taking cannabidiol or fenfluramine decreases over time in DS patients on stiripentol, while the percentage increases in non-DS patients from initiation. This new analysis can supplement the current label and provide guidance for dosing and use with concomitant medications to US prescribers of stiripentol.

Funding: Biocodex, Inc.