Fintepla (low-dose fenfluramine) / UCB, Nippon Shinyaku - LARVOL DELTA

Characterizing SCN1A -Related Disorders Using Real-World Data Across 681 Patient-Years. (PubMed, medRxiv) - "Changes in treatment coincided with the diagnosis of an SCN1A -related disorder, with an increase in cannabidiol, clobazam, and fenfluramine and reduction in sodium channel-blocker use following genetic diagnosis. In summary, we reconstructed the longitudinal disease history of SCN1A -related disorders from electronic medical records using a standardized framework for the analysis of real-world clinical data. We refine existing natural history data of SCN1A -related disorders by providing a granular landscape of seizures, comorbidities, and treatment approaches over time."

Journal • Real-world evidence • Ataxia • CNS Disorders • Epilepsy • Movement Disorders

Recent advances in PET measures of brain 5-HT release. (PubMed, J Cereb Blood Flow Metab) - "These include the use of agonist radioligands with preferential binding to high-affinity receptor states, such as [11C]AZ10419369 and [11C]Cimbi-36, antagonist tracers, such as [18F]Altanserin, refined challenge designs using pharmacological 5-HT releasers, such as fenfluramine and amphetamine, and the integration of hybrid PET/MR imaging to assess neurovascular aspects. These advances have shifted the field from questioning feasibility to addressing optimal strategies for measuring serotonergic dynamics."

Journal • Review • CNS Disorders • Mental Retardation • Psychiatry

Practical consensus recommendations for polytherapy involving stiripentol in Dravet syndrome: A nominal group approach. (PubMed, Epilepsia Open) - "Dravet syndrome is a rare and severe form of epilepsy that begins in early childhood, with seizures that often persist throughout life. Because the disease is highly drug resistant, most patients are treated with polytherapy, making clinical management complex for clinicians. This consensus brings together published evidence and expert experience to provide practical guidance on the management of polytherapy in Dravet syndrome, supporting clinicians in optimizing patient care."

Journal • CNS Disorders • Epilepsy

Prevalence, Treatment Patterns, and Healthcare Resource Utilization Among Patients with CDKL5 Deficiency Disorder: Retrospective Analysis of US Claims Data (AAN 2026) - "Levetiracetam (34%), clobazam (33%), and clonazepam (30%) were the most common seizure-related drugs utilized. Increase in uptake post diagnosis was seen for ganaxolone (23%), fenfluramine (5%), and cenobamate (4%)... This real-world retrospective analysis reports prevalence for patients with CDD for the first time in a large, claims-based population. Comorbidities and HCRU burden are significant for patients with CDD."

HEOR • Retrospective data • Cerebral Palsy • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Movement Disorders • CDKL5

Assessing the Potential Cardiovascular Risk of Microdosing the Psychedelic LSD in Mice. (PubMed, ACS Pharmacol Transl Sci) - "We calculated that levels of 5-HT2B activation by low-dose LSD were substantial, but short-lived, compared to the cardiotoxin d-fenfluramine. Together, these data provide no evidence of ventricular or valvular remodeling associated with prolonged administration of low-dose LSD in mice."

Journal • Preclinical • Cardiovascular • Heart Failure

Fenfluramine in CDKL5 Deficiency Disorder: Primary Efficacy and Safety Results from a Phase 3, Randomized, Double-blind, Placebo-controlled Study (AAN 2026) - P3 | "In this study, fenfluramine provided significantly greater CMSF reduction in patients with CDD versus placebo; global functioning was improved and TEAEs were consistent with known fenfluramine safety profile. Fenfluramine may be a promising therapy for treating CDD-associated seizures."

Clinical • P3 data • Cardiovascular • CNS Disorders • Epilepsy • Genetic Disorders • Heart Failure • Pulmonary Arterial Hypertension • Respiratory Diseases • CDKL5

Stratified Effectiveness and Safety of Fenfluramine in Dravet and Lennox-Gastaut Syndromes: A Retrospective Comparative Analysis of Trial and Open-label Extension Data (2018-2024) (AAN 2026) - "Results were descriptively compared across DS and LGS by age (<6 y, 6-17 y. ≥18 y) and stiripentol (STP) co-therapy by using IBMSPSS V 29. Fenfluramine provides durable seizure reductions and consistent safety across DS and LGS, with the strongest benefit observed in younger and STP-naïve patients. The absence of cardiac toxicity across ~1,000 patient-years supports its long-term safety. Stratified synthesis of multi-trial evidence enables precision use of FFA in developmental and epileptic encephalopathies."

Retrospective data • Cardiovascular • CNS Disorders • Epilepsy • Heart Failure • Pulmonary Arterial Hypertension • Respiratory Diseases • CSF1

Healthcare Resource Utilization, Antiseizure Medication Claims, and Treatment Persistence in Patients with Lennox-Gastaut Syndrome Receiving Fenfluramine in the United States (AAN 2026) - "Real-world claims data of patients with LGS receiving fenfluramine showed significant reductions in HCRU and ASM burden and good treatment persistence at 6 and 12 months."

Clinical • HEOR • CNS Disorders • Epilepsy

Association of Fenfluramine Treatment and Everyday Executive Functioning in Adult Patients with Lennox-Gastaut Syndrome (AAN 2026) - P3 | "Adults with LGS treated with fenfluramine had clinically meaningful EEF improvements during a 14-week RCT and its 12-month OLE. Reductions in seizures associated with a fall and EEF improvements were partially independent of each other."

Clinical • CNS Disorders • Epilepsy

Industry Therapeutic Update from UCB: Scientific exchange on new clinical and real-world evidence for FINTEPLA (fenfluramine) in Dravet and Lennox-Gastaut syndromes (AAN 2026) - "The program will also highlight real-world use of an updated refractory epilepsy screening tool to support identification of Lennox-Gastaut syndrome and conclude with an interactive Q&A. OBJECTIVES- Characterize the long-term tolerability and global functioning outcomes associated with fenfluramine in patients with Dravet and Lennox-Gastaut syndromes- Describe the association between fenfluramine and everyday executive functioning in adults with Lennox-Gastaut syndrome- Review real-world healthcare resource utilization and antiseizure medication patterns in patients with Lennox-Gastaut syndrome treated with fenfluramine- Explore the role of an updated refractory epilepsy screening tool in identifying Lennox-Gastaut syndrome in real-world practice"

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

Strong Execution Fueling Sustained Company Growth (UCB Press Release) - "Revenue in 2025 increased to € 7 741 million (+26%; +29% CER1) and net sales went up to € 7 388 million (+32%; +35% CER1). This growth was driven by the strong, consistent growth of UCB’s growth drivers: BIMZELX, EVENITY, FINTEPLA, RYSTIGGO and ZILBRYSQ, thanks to strong execution."

Sales • Ankylosing Spondylitis • CNS Disorders • Hidradenitis Suppurativa • Myasthenia Gravis • Osteoporosis • Psoriasis • Psoriatic Arthritis

Fenfluramine in SCN1A-related GEFS+: A multicenter observational study on efficacy, EEG improvement, and tolerability. (PubMed, Epilepsia Open) - "EEG recordings improved, and caregivers reported better alertness in some patients. Treatment was generally well tolerated, with only mild, temporary side effects."

Journal • Observational data • CNS Disorders • Epilepsy

Use of fenfluramine in MECP2-related Rett syndrome: Findings from a retrospective multicenter pediatric case series. (PubMed, Epilepsy Behav) - "FFA appears effective and generally well-tolerated in patients with RTT and drug-resistant epilepsy, particularly for generalized tonic-clonic seizures. Cognitive and behavioral improvements reported by caregivers may be attributable to a combination of serotonergic receptor modulation and reduced seizure burden. Despite polytherapy, side effects were minimal. These findings align with existing literature on FFA use in other developmental epileptic encephalopathies. Prospective studies on larger cohorts with long-term monitoring are needed to validate efficacy, safety, and cognitive-behavioral outcomes."

Journal • Retrospective data • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Movement Disorders • Pediatrics • Psychiatry

Ameliorating Seizures in Dravet Syndrome: A Review of Newly Approved and Investigational Drugs, RNA and Gene-Based Therapies. (PubMed, CNS Drugs) - "Recent therapeutic developments include the approval of new antiseizure medications such as fenfluramine and pharmaceutical-grade cannabidiol, which have demonstrated efficacy and tolerability in randomised placebo-controlled trials...Antisense oligonucleotides (e.g., STK-001) aim to restore SCN1A expression, while emerging gene therapy approaches, including engineered AAV vectors (e.g., ETX101) and CRISPR-mediated transcriptional activation, seek to directly modify disease biology...Future directions include defining the most effective genetic therapies to improve outcomes, and ideally cure, all features of DS; optimal timing to deliver interventions as well as benefits derived from administration at later ages; ideal combinations of therapies; comparison of outcomes of targeted therapies with natural history studies and biomarker development. Together, these advances signal a paradigm shift in epilepsy management from symptomatic treatment to precision medicine for..."

Journal • Review • CNS Disorders • Epilepsy • Gene Therapies • NAV1

Flexibility in the Modelling of Comparative Effectiveness in the Absence of Head-to-Head Comparisons in the NICE Single Technology Appraisal of Fenfluramine for Treating Seizures Associated with Lennox-Gastaut Syndrome: An External Assessment Group Perspective. (PubMed, Pharmacoeconomics) - No abstract available

Head-to-Head • HEOR • Journal • CNS Disorders • Epilepsy

Efficacy of Dravet Syndrome Treatments in a Subset of Individuals with 2q24.3 Deletion: A-5 Patient Case Series. (PubMed, J Child Neurol) - "We found evidence of efficacy for valproic acid, clobazam, and cannabidiol whereas levetiracetam and phenobarbital were not beneficial. Additional studies are necessary to examine the efficacy of fenfluramine and the ketogenic diet."

Journal • CNS Disorders • Epilepsy

From Symptomatic Therapies to Disease-Modifying Approaches for Neuronal Sodium Channel Disorders. (PubMed, Int J Mol Sci) - "Additionally, pharmacological agents such as fenfluramine, stiripentol, and cannabidiol, although not acting directly on sodium channels, represent recognized therapeutic options for SCN1A-related Dravet syndrome. This review summarizes recent advances in approved and investigational treatments for sodium channel-related neurological disorders, highlighting the transition from symptomatic to precision therapies."

Journal • Review • CNS Disorders • Epilepsy • Gene Therapies

Safety of Drugs Used in Difficult-to-Treat Epileptic Syndromes: A Disproportionality Analysis Using the Eudravigilance Database. (PubMed, Pharmaceuticals (Basel)) - " We retrospectively analyzed the publicly available data regarding Individual Case Safety Reports (ICSRs), presenting stiripentol, cannabidiol, or fenfluramine as suspected drugs, reported on the Eudravigilance database until the third quarter of 2024. Our analysis did not highlight new and unexpected serious safety signals but confirmed the need to strictly monitor patients for the risk of adverse events. However, further prospective studies are required to better clarify the safety profile of these drugs in order to optimize their use."

Journal • Cardiovascular • CNS Disorders • Epilepsy

Fenfluramine for seizures associated with Dravet syndrome and Lennox-Gastaut syndrome. (PubMed, Aust Prescr) - No abstract available

Journal • Review • CNS Disorders • Epilepsy

Cost-effectiveness of fenfluramine as add-on treatment in the management of Dravet Syndrome: A real-world multicenter study. (PubMed, Epilepsia Open) - "Dravet syndrome is a severe and rare type of epilepsy that can be very hard on patients and their families. It also puts a great burden on healthcare systems. A new treatment, fenfluramine (FFA), helps better control seizures. Although FFA increases drug treatment costs, this cost is actually balanced out by reduced emergency visits and hospital stays for severe seizures, leading to overall lower healthcare expenses."

Clinical • HEOR • Journal • Real-world evidence • CNS Disorders • Epilepsy • Rare Diseases

Fenfluramine for late-onset epileptic spasms. (PubMed, Epileptic Disord) - No abstract available

Journal • CNS Disorders • Epilepsy

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

Reply to Chen et al. "A Postmarketing Pharmacovigilance Study of Fenfluramine: Adverse Event Data Mining and Analysis Based on the US Food and Drug Administration Public Data Open Project (openFDA)". (PubMed, Pediatr Neurol) - No abstract available

Adverse events • Journal • P4 data

Association of Fenfluramine Treatment and Everyday Executive Functioning in Adult Patients With Lennox-Gastaut Syndrome (AES 2025) - P3 | "In this post hoc analysis, clinically meaningful improvements in EEF were seen in adults with LGS treated with FFA in the RCT; consistent improvements were seen in the 12-mo OLE. There were no strong correlations between reductions in seizures associated with a fall and improvements in EEF, suggesting that these outcomes were partly independent of each other."

Clinical • Late-breaking abstract • CNS Disorders • Developmental Disorders • Epilepsy • Mental Retardation

Healthcare Resource Utilization and Antiseizure Medication Claims in Patients With Lennox-Gastaut Syndrome Receiving Fenfluramine in the United States (AES 2025) - "FFA use in patients with LGS was associated with reductions in ER visits (all-cause and seizure-related), inpatient hospitalizations (all-cause and seizure-related), and ambulance use. The results for ER visits and ambulance use remain robust, following more rigorous analytical approaches. The average number of all ASM claims and unique ASM claims per patient decreased following FFA initiation, suggesting that treatment with FFA reduces the total ASM drug load in patients with LGS."

Clinical • HEOR • Late-breaking abstract • CNS Disorders • Epilepsy