Diacomit (stiripentol) / Biocodex, Meiji Seika - LARVOL DELTA

Relieving the Weight: Fenfluramine's Re-emergence as Antiseizure Medication for Lennox-Gastaut and Dravet Syndrome. (PubMed, Ann Pharmacother) - "As seen through the results of multiple clinical trials, fenfluramine can provide significant seizure reduction for Lennox-Gastaut and Dravet patients with treatment-resistant seizures. Risks can be mitigated through adherence to proper programs and schedules, and can provide enough benefits to outweigh risks in patients with particularly resistant epilepsies."

Journal • Review • Cardiovascular • CNS Disorders • Epilepsy • Pediatrics

Stiripentol use in Dravet syndrome patients in the USA: Results of a real-world study. (PubMed, Epilepsia Open) - "The STIRUS study found that stiripentol, an anti-seizure medication specifically approved for Dravet Syndrome, helped reduce seizures and hospital visits for children living with this severe form of epilepsy. Patients had fewer convulsive seizures, needed fewer rescue medicines, and reported better quality of life. Despite these benefits, stiripentol is still not widely used and often started too late in treatment, suggesting earlier use could further improve outcomes."

Journal • Real-world evidence • CNS Disorders • Epilepsy

Physiologically Based Pharmacokinetic Modeling of Clobazam and Stiripentol Co-Therapy in Dravet Syndrome. (PubMed, J Pers Med) - "Extrapolation to pediatric patients under two years of age predicted CLB, N-CLB, and STP exposures that were comparable to older children and remained within their reported efficacy and safety margins, suggesting no major ontogeny-related effect on exposure. The PBPK model supports the safe extrapolation of CLB and STP co-administration to pediatric Dravet syndrome patients as young as six months."

Journal • PK/PD data • CNS Disorders • Epilepsy • Pediatrics • CYP2C19

Stiripentol Use in Lennox-Gastaut Syndrome: Results from a Phase 2 Clinical Trial (AES 2025) - "They received an average of 3 ASMs, most commonly valproate (n=10) and clobazam (n=8). Despite the small sample size, stiripentol appeared to reduce seizure burden in LGS patients. These promising results support further investigation in a larger, controlled trial."

Clinical • Late-breaking abstract • P2 data • Absence Seizure Disorder • CNS Disorders • Epilepsy • Gastroenterology • Gastrointestinal Disorder

Stiripentol Use in Dravet Syndrome Is Associated with Lower Mortality Rates in a Real-World United States (US) Cohort (AES 2025) - "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."

Clinical • Late-breaking abstract • Real-world • Real-world evidence • CNS Disorders • Epilepsy

Efficacy and Tolerability of Stiripentol Across non-Dravet Developmental and Epileptic Encephalopathies: A Literature Review Including Genetic and Syndromic Epilepsies (AES 2025) - "STP demonstrates consistent efficacy and acceptable tolerability across a wide spectrum of non-Dravet DEEs, including ultra-rare genetic forms. Its effectiveness across seizure types and in RSE supports its consideration as a treatment option in complex drug-resistant epilepsy. Prospective, genotype-driven studies are warranted to better define the optimal use of STP in DEEs."

Clinical • Review • Anorexia • Ataxia • CNS Disorders • Epilepsy • Movement Disorders

Real-World Utilization of Stiripentol in Children Aged 3 Years and Younger: A US Perspective (AES 2025) - "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)... 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."

Clinical • Real-world • Real-world evidence • CNS Disorders • Epilepsy

Practical Consensus Recommendations for Polytherapy Involving Stiripentol in Dravet Syndrome: A Nominal Group Approach (AES 2025) - "The therapeutic arsenal includes non-specific first-line treatments (valproate and clobazam), specific therapies including stiripentol (STP), cannabidiol (CBD) fenfluramine (FFA), and other ASMs such as bromide, topiramate and levetiracetam. This expert consensus, developed through a nominal group technique and supported by an independent vote from international clinicians, provides a framework for all physicians managing DS to evaluate and refine their polytherapy practices with the goal of improving patient care."

CNS Disorders • Epilepsy

Final Results From a Long-Term Open-Label Extension Study (Up to 4 Years): Tolerability of Fenfluramine and Global Functioning of Pediatric and Adult Patients With Dravet or Lennox-Gastaut Syndromes (AES 2025) - P1, P3 | "Last FFA doses from the prior studies were continued, then flexibly titrated as needed (max FFA 0.7 mg/kg/d [26 mg/d] without stiripentol [STP] or FFA 0.4 mg/kg/d [17 mg/d] with STP). In this OLE study of patients with DS or LGS, TEAEs were consistent with the known FFA safety profile, and no new or unexpected safety signals were observed. Investigator and caregiver CGI–I ratings suggested sustained clinical benefit (median overall FFA exposure = 4 years). These data support long-term FFA use in pediatric and adult patients with DS or LGS."

Clinical • Late-breaking abstract • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Heart Failure • Pediatrics • Pulmonary Arterial Hypertension • Respiratory Diseases

Need for Reintroduction of Stiripentol After Weaning in Patients with Dravet Syndrome: A Multicenter Case Series (AES 2025) - "We aimed to evaluate polypharmacy management in DS individuals treated with stiripentol (STP), an approved adjunctive therapy to clobazam (CLB) in the US, and to CLB and valproate (VPA) in Europe. Ten patients with DS from six centres who underwent STP weaning followed by reinstitution of STP were retrospectively ascertained, and descriptive analysis performed. STP was introduced in all patients for seizure control despite multiple prior ASM attempts (median 3 [range 1-7, n=9], most commonly VPA [88.9% patients], topiramate [55.6%], CLB [44.4%])...STP was tapered or discontinued after a median treatment duration of 102 months (range 17-203) for (i) AEs with add-on fenfluramine (FFA) (unsteadiness, tiredness and weight loss, myoclonic seizure cluster and moderate anorexia, severe psychiatric disorder, n=3), (ii) FFA clinical trials or dose increase (n=4), (iii) attempts to simplify polytherapy after seizure reduction with add-on FFA (n=2), cannabidiol (n=2) or ketogenic..."

Clinical • Late-breaking abstract • Absence Seizure Disorder • Anorexia • CNS Disorders • Constipation • Epilepsy • Gastroenterology • Gastrointestinal Disorder • Mental Retardation • Movement Disorders • Psychiatry

Practical Consensus Recommendations for Rational Polytherapy Involving Stiripentol in Dravet Syndrome: Preliminary results of a US Cohort (AES 2025) - "Consensus recommendations1 highlight valproic acid as 1st line treatment, stiripentol (STP), fenfluramine (FFA), and/ or clobazam (CLB) as 1st or 2nd line and other ASMs including cannabidiol (CBD), levetiracetam (LEV) and topiramate (TPM) as 3rd and 4th line. The preliminary results of the expert consensus on DS treatment, developed through a nominal group and validated by independent voting among US clinicians, provide a practical framework for US providers managing DS to help guide polytherapy decisions. The initial results reinforce the infrequent use of DS-specific ASMs highlighted in previous reviews.2 While there was a strong consensus supporting STP's efficacy when added to an existing ASMs, the data revealed limited provider confidence in the management of these DS-specific ASMs, including STP, underscoring the need for targeted education."

Late-breaking abstract • CNS Disorders • Epilepsy

Utilization and Prescribing Patterns of Stiripentol for Dravet Syndrome: Preliminary Results of an International Physician Survey (AES 2025) - "Rationale: Stiripentol (STP) is approved for the treatment of seizures associated with Dravet Syndrome (DS) in patients taking clobazam who are 6 months of age and older...64.3% co-prescribed sodium valproate. Half prescribed STP in conjunction with fenfluramine (FFA), 75.0% with cannabidiol (CBD) with most (85.7%) adjusting the FFA dose when used in combination.When initiating therapy, providers prioritized seizure frequency (53.6%), seizure length (35.7%), and seizure character (10.7%)... Preliminary findings from this survey highlight a modest uptick in STP utilization in DS across experienced pediatric epilepsy specialists. While most providers reported experiencing clinical benefit, adverse effects, and insurance-related barriers were common. These findings suggest STP is utilized but variably implemented in practice, with opportunities to refine dosing strategies and address systemic barriers to optimize outcomes for patients with DS."

Late-breaking abstract • Anesthesia • CNS Disorders • Epilepsy

Real World Utilization of Stiripentol (STP) by United States (US) Prescribers: A 3-Year Analysis Update (AES 2025) - "Despite rational polytherapy recommendations and availability of DS-approved antiseizure medications (ASMs), stiripentol (STP), cannabidiol (CBD), and fenfluramine (FFA), integration into treatment regimens remains limited1...At STP initiation, 71% were taking clobazam (CLB), 33% CBD, and 21% on FFA (previously 75%, 37%, 24%)...Levetiracetam (16%) and valproic acid (15%) were the most common non-DS approved ASMs... This 3-year analysis shows STP dosing remained consistent and below the FDA-approved dose of 50 mg/kg/day, with higher doses in younger patients. Doses of STP increased with longer treatment duration, reflecting titration to maintenance dosing, which is critical for tolerability and reduced premature discontinuations. While maintenance doses in younger patients were consistent with approved dosing, patients ≥ 6 years received lower average STP doses even after 8+ months on therapy."

Clinical • Late-breaking abstract • Real-world • Real-world evidence • CNS Disorders • Epilepsy

Underutilization of FDA approved Dravet Syndrome Specific Therapies: Findings from a US Multi-Center Survey and Advisory Board (AES 2025) - "Despite the availability and international consensus recommendations for DS-approved antiseizure medications (ASMs), stiripentol (STP), fenfluramine (FFA), and cannabidiol (CBD), a recent analysis found that these ASMs have been underutilized in DS patients, with only 7% of patients prescribed STP, 16% FFA, and 28% CBD.1,2 A US multi-center survey and advisory board aimed to explore ASM selection, barriers to DS-specific ASM use, and strategies to improve alignment with international consensus recommendations.2 A voluntary survey was completed by 38 clinicians from 27, Level IV National Association of Epilepsy Centers (NAECs)... The survey identified valproate (47%) or clobazam (CLB) (24%) as the most common 1st line ASMs considered when a patient is diagnosed with DS, with levetiracetam as a common ASM already included in a patient's regimen... This analysis confirms underutilization of DS-approved ASM therapies in DS management, highlighting a gap between..."

Clinical • Late-breaking abstract • CNS Disorders • Epilepsy

Real-World Use and Effectiveness of Stiripentol in U.S. Patients with Dravet Syndrome: Results from the STIRUS Study (AES 2025) - "At baseline, patients were taking a median of 3 antiseizure medications, most commonly clobazam (n=80), cannabidiol (n=45), valproate (n=42), fenfluramine (n=24), or levetiracetam (n=23). In this contemporary U.S. DS cohort, the use of stiripentol was associated with a reduction in seizure burden, status epilepticus episodes, and healthcare utilization, with consistent benefits observed across age groups and co-medication profiles. These findings support STP as an effective treatment option beyond its approved use with clobazam."

Clinical • Real-world • Real-world evidence • CNS Disorders • Epilepsy

The SCN1A-Horizons study: Baseline report of a prospective longitudinal natural history study of SCN1A-related epilepsies in the United Kingdom (UK) (AES 2025) - "Median seizure onset age was 5 months (range 2-78), and most frequent subsequent seizure type was generalised tonic-clonic (109; 90.8%).Median number of current ASMs was 3 (range 0-5); most commonly sodium valproate (83; 69.2%), clobazam (67; 55.8%), stiripentol (55; 45.8%) and fenfluramine (36; 30.0%). The number of previous ASMs used ranged from 0-14 (median=1), most frequently levetiracetam (48; 40.0%)... These baseline findings demonstrate the high burden of seizures and comorbidities amongst individuals with SCN1A-related epilepsy. Ongoing follow-up of this cohort will inform care standards and treatment strategies."

Clinical • ADHD (Impulsive Aggression) • Attention Deficit Hyperactivity Disorder • Autism Spectrum Disorder • CNS Disorders • Epilepsy • Psychiatry • Sleep Disorder

Long-term Effectiveness of Stiripentol in Reducing Seizure Burden and Status Epilepticus in Dravet Syndrome: Results from a 3-Year Japanese Post-Marketing Surveillance (AES 2025) - "Stiripentol demonstrated sustained long-term effectiveness in reducing multiple seizure types and status epilepticus over a 3-year period, with an acceptable safety and tolerability profile. Early initiation of STP treatment (< 3 years) was associated with greater clinical benefit and improved tolerability, underscoring the importance of prompt therapeutic intervention in Dravet syndrome."

P4 data • Absence Seizure Disorder • CNS Disorders • Epilepsy

Industry Supported Scientific Exhibit | Stiripentol: Data Advances and Clinical Applications - A Global Perspective (AES 2025) - "Sponsored by Biocodex, Inc"

Clinical

SIG | Neuropharmacology: Use of Anti-seizure Medications in the Era of AI and Precision Medicine (AES 2025) - "Overview There have been several new anti-seizure medications (ASM) FDA-approved for treatment of epilepsy in the last 5-6 years (for example: Stiripentol, Fenfluramine, Ganaxolone, Cenobamate). The 2025 Neuropharmacology SIG addresses the mechanisms of action of these new medications, use of EMR-integrated AI to guide the use of ASMs in different clinical scenarios, detection of drug-drug interactions, and use of ASMs in epilepsy treatment from a precision medicine approach. Following the presentations, a moderated panel discussion addresses questions from the audience.Learning Objectives Following participation in this activity, participants will be able to: • Identify mechanisms of action and clinical indications of newly FDA-approved anti-seizure medications • Understand advantages and challenges of AI models for identifying drug-drug interactions in epilepsy, enhancing patient safety and treatment efficacy • Describe use of anti-seizure medications from a precision..."

CNS Disorders • Epilepsy

EPIPOP: Population Pharmacokinetics of Antiepileptic in Pediatrics (clinicaltrials.gov) - P=N/A | N=753 | Completed | Sponsor: Assistance Publique - Hôpitaux de Paris | Recruiting ➔ Completed

Trial completion • CNS Disorders • Epilepsy • Pediatrics

Hypoxia-Induced Histone Lactylation Drives Cisplatin Resistance in Bladder Cancer by Promoting RBM15-Dependent m6A Methylation of IGFBP3. (PubMed, Research (Wash D C)) - "Targeting this axis with LDHA inhibitor (stiripentol) and EGFR inhibitor (gefitinib) synergistically reversed cisplatin resistance in vitro and in vivo. This study unveils the "hypoxia-H3K18la-RBM15-IGFBP3" axis as a central driver of cisplatin resistance and proposes dual metabolic-epigenetic inhibition as a therapeutic strategy for refractory BCa."

Journal • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • IGFBP3 • LDHA • RBM15

α-Asaronol, a Low-Toxicity α-Asarone Metabolite, Suppresses Seizures in Zebrafish Dravet Syndrome via Positive Modulation of GABAA Receptors and LDH Inhibition. (PubMed, Neuropharmacology) - "Notably, α-AOL exhibited superior efficacy in scn1Lab-/- mutants compared to α-asarone and reference drugs stiripentol and cannabidiol. Mechanistic studies identified dual pathways for α-AOL: potentiation of GABAA receptor currents (EC50 = 34.0 μmol/L) and inhibition of neuronal lactate dehydrogenase activity. These findings establish terminal oxidation of α-asarone as a critical metabolic pathway enhancing anti-seizure efficacy while reducing toxicity, positioning α-AOL as a promising lead compound for DS therapeutics."

Journal • CNS Disorders • Epilepsy • Pediatrics

Clinical and genetic landscape of epilepsies with absence seizures and single-gene etiology. (PubMed, Epilepsia) - "Absence seizures are a common manifestation of different monogenic epilepsies, often associated with early onset, atypical clinical and/or EEG features, developmental delay or drug resistance. Classification models should incorporate genetic data alongside electroclinical features, especially as next-generation sequencing is increasingly used."

Clinical • Journal • Absence Seizure Disorder • CNS Disorders • Developmental Disorders • Epilepsy • Mental Retardation • CHD2 • SLC2A1 • STARD9

Stiripentol-loaded self-nanoemulsifying delivery system for glioblastoma: therapeutic efficacy in 3D spheroid and xenograft model. (PubMed, Nanomedicine (Lond)) - "STP SNEDDS showed improved water solubility and drug release profile, stability, relative safety in normal cells and brain tissue, and decreased 3D spheroid growth, tumor volume, tumor weight, and proliferative marker Ki67. This work highlights the potential of STP SNEDDS as a potential therapeutic for GBM."

Journal • Preclinical • Brain Cancer • Glioblastoma • Oncology • Solid Tumor

STIRIPENTOL AS A POTENTIAL THERAPEUTIC OPTION IN PRIMARY HYPEROXALURIA TYPE 3: A CASE REPORT (ESPN 2025) - "This case suggests stiripentol may effectively reduce urinary oxalate excretion in PH3, providing a safe and effective therapeutic option. Further studies are warranted to assess its long-term efficacy and safety in this patient population."

Case report • Clinical • CNS Disorders • Epilepsy • Metabolic Disorders • Nephrology • Renal Calculi • Renal Disease • Urinary Incontinence