vigabatrin / Generic mfg. - LARVOL DELTA

Reassessing vigabatrin in infantile spasms: clinical risks, mechanistic insights, and future directions. (PubMed, Pediatr Res) - No abstract available

Journal • CNS Disorders • Epilepsy

Genetic Etiology of Infantile Spasms in Peruvian Children: A Multicenter Study. (PubMed, J Child Neurol) - "Vigabatrin was the most frequently used control medication (54.55%, n = 30)...A wide range of genetic variants was found in one-third of the cases. Prompt genetic identification in IESS is recommended to optimize treatment and improve the prognosis."

Clinical • Journal • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Pediatrics

The Enigma of West Syndrome: A Case of Infantile Spasms Without Genetic Clues. (PubMed, Case Rep Pediatr) - "Current treatment recommendations by pediatric neurologists in the GCC include first-line therapy with vigabatrin alone or in combination with high-dose oral corticosteroids...This case highlights the challenges in diagnosing and managing West syndrome, especially with inconclusive genetic analysis. It emphasizes the need for continued advancements in genetic diagnostics to uncover new mutations and improve patient outcomes."

Journal • Cardiovascular • CNS Disorders • Epilepsy • Hypertension • Infectious Disease • Mood Disorders • Pediatrics

A potential explanation of the pathophysiology of vigabatrin-associated brain abnormalities on MRI. (PubMed, Pediatr Res) - No abstract available

Journal

Molecular docking and In-Silico pharmacokinetic analysis of 1,3-benzothiazole-2-amine derivatives as anticonvulsants. (PubMed, J Taibah Univ Med Sci) - "Compared with the reference medication vigabatrin (-5.2 kcal/mol), seven analogues (A1, A3, A9, A10, A11, A12, and A14) had higher binding affinities (-5.9, -5.8, -6.1, -5.9, -6.0, -6.1, and -6.6 kcal/mol, respectively) for GABA-AT binding. NavMs binding analysis showed that only analogue A14 had a binding affinity (5.0 kcal/mol) comparable to that of the reference medication lamotrigine. In-silico investigations identified benzothiazole compounds with high anticonvulsant properties as suitable candidates for synthesis and pharmacological testing."

Journal • PK/PD data • Review • CNS Disorders • Epilepsy • Infectious Disease • Oncology • Pain

OV329, a Potent Next Generation GABA-AT Inhibitor, is Well-tolerated and Produces GABA-ergic Cortical Inhibition in a Phase 1 SAD/MAD Study in Healthy Volunteers (AES 2025) - "Exploratory biomarkers assessed potential PD effects, including transcranial magnetic stimulation (TMS) metrics that were previously used to study a 1st-generation GABA-AT inhibitor, vigabatrin (VGB). OV329 is safe and well tolerated across the tested dose range. TMS measures indicate, like VGB, OV329 augments GABA- mediated TMS measures of cortical inhibition, and is a potential anti-convulsant candidate for patients with epilepsy supporting further clinical studies"

Clinical • Late-breaking abstract • P1 data • CNS Disorders • Epilepsy • Ophthalmology

An Infantile Epileptic Spasms Syndrome Multidisciplinary Program: One Center's Experience. (AES 2025) - "We have streamlined the initiation of 3 first-line treatments (ACTH, High-dose oral corticosteroids, vigabatrin) during hospitalization, and made electronic medical record dot phrases for the residents...We have demonstrated the importance of a multidisciplinary practice to improve clinical care: 1) Repeating a brain MRI due to focal semiology, finding a focal cortical dysplasia, and seizure freedom after resective surgery 2) Partnering with Endocrinology to safely manage a prolonged 2 month prednisolone wean followed by a 7 week hydrocortisone wean resulting in seizure freedom 3) Collaborating with research in a patient with pathogenic variant in CAMK2A, functional studies revealed a gain of function of the calmodulin, and treated with Depakote to reduce calmodulin activity... We have established an efficient, organized and reliable IESS program with several strengths. We communicate closely with the PCPs, follow a multidisciplinary approach, have reserved EEG slots,..."

Cardiovascular • CNS Disorders • Endocrine Disorders • Epilepsy • Genetic Disorders • Hypertension • Nephrology • CAMK2A • XBP1

Corpus Callosotomy for Treatment of Infantile Epileptic Spasms Syndrome: The UCLA Experience (AES 2025) - "Median number of prior anti-seizure medications was 8 (IQR 7 – 10), and all subjects failed to respond to a trial of standard first-line therapy (prednisolone, ACTH, vigabatrin, or a combination thereof)...Patient 2 achieved seizure-freedom (now >1 yr) in response to cannabidiol and clobazam, prior to proposed surgical resection...After relapse with tonic seizures and epileptic spasms, both patients have since responded to cenobamate and remain seizure-free > 1yr... This study suggests that corpus callosotomy may aid in treatment of children with IESS, though the response rate in this series is lower than prior reports (Baba et al, 2018). Nevertheless, CC appears to be helpful in a substantial minority of patients, and CC should perhaps be considered much earlier in the course of epilepsy. Further study is clearly warranted to identify best candidates for CC in the setting of IESS."

CNS Disorders • Epilepsy

Evaluation of Pharmacological Treatments for Infantile Spasms: A Review of ClinicalTrials.gov (AES 2025) - "Key elements extracted included therapeutic agents, study design, primary endpoints, and reported efficacy and safety outcomes. The included trials assessed a range of pharmacologic agents, spanning both established therapies (ACTH, vigabatrin, prednisolone) and investigational compounds (cannabidiol, ganaxolone, TAK-935, tricaprilin, pyridoxine, lithium carbonate). This review highlights the diversity of pharmacologic interventions under investigation for IS and reinforces the role of both established and emerging therapies. While completed trials provide valuable insight into evolving treatment strategies, important gaps remain—particularly the lack of patient-level outcomes and long-term neurodevelopmental data. Future research should focus on stratified study designs, biomarker-informed approaches, and extended follow-up to optimize treatment selection and improve clinical outcomes in this high-risk pediatric population."

Clinical • Review • CNS Disorders • Epilepsy • Gastrointestinal Disorder

Association between Infantile Epileptic Spasms Syndrome and Autism. (AES 2025) - " Using single-center (UCLA) data housed within the Pediatric Epilepsy Learning Healthcare System (PELHS) infrastructure, we defined IESS as presence of an ICD diagnostic code and prescription of at least one standard therapy (prednisolone, ACTH, or vigabatrin)... Automated EMR abstraction can reliably identify IESS and autism diagnoses and approximate key treatment parameters. However, precise dating of IESS onset and response still benefits from manual review or advanced NLP methods. Scaling this approach to multi-center datasets may clarify the mechanistic link between IESS severity and subsequent autism risk."

Autism Spectrum Disorder • CNS Disorders • Epilepsy • Genetic Disorders

Evaluation of ASM with new mechanisms of action in the GAERS model of absence seizure (AES 2025) - "Indeed, SWD in GAERS are inhibited by the anti-seizure medications (ASM) used to treat absence seizures, such as ethosuximide, valproate and lamotrigine. But SWD in GAERS are also aggravated by ASM or other drugs known for worsening absence in patients, e.g. carbamazepine or vigabatrin.Some recent compounds with antiseizure properties and specific mechanisms of action have never been evaluated in the GAERS...Retigabine (4-8 mg/kg PO) produced a significant aggravation of SWD... Evaluation of new mechanisms of action in the GAERS may provide fresh hypothesis for the development of new compounds addressing absence seizures. Our study indicates that targeting potassium channels may be at risk of aggravating SWD, whereas targeting the 5-HT system may produce an interesting effect on absence seizures."

Absence Seizure Disorder • Anesthesia • CNS Disorders • Epilepsy

Blarcamesine (ANAVEX®2–73) Attenuates Spasms and Hypsarrhythmia in a Rat Model of Infantile Epilepsy Spasms Syndrome (AES 2025) - "Current FDA-approved management including adrenocorticotropic hormone (ACTH) and vigabatrin, demonstrate efficacy in only 50–60% of cases and often carry significant side effects, such as immunosuppression and visual field deficits. These results highlight the efficacy of blarcamesine to suppress spasms, ameliorate EEG abnormalities, and prevent progression to spontaneous seizures later during development, advancing approaches for managing IESS."

Preclinical • Absence Seizure Disorder • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • CDKN1A

Vigabatrin Treatment Prior To Seizures Decreased the Proportion of EEGs with Epileptiform Activity in Infants with Tuberous Sclerosis Complex During the PREVeNT Trial (AES 2025) - "Treatment with vigabatrin at first detection of epileptiform activity on an EEG decreased by ~10% the overall proportion of EEGs with epileptiform activity up to 3 years of age. Patients in the early vigabatrin treatment group without epileptic spasm drove the reduction in the frequency of epileptiform EEGs . Early vigabatrin did not impact the number of epileptiform EEGs in subjects eventually developing epileptic spasms."

CNS Disorders • Epilepsy

Long-term Outcomes of Preventive Antiseizure Treatment in TSC patients – Real World Data. (AES 2025) - "Preventive treatment with vigabatrin in infancy improves long-term neuropsychiatric and epilepsy outcomes in children with TSC."

Clinical • Real-world • Real-world evidence • Autism Spectrum Disorder • CNS Disorders • Developmental Disorders • Epilepsy • Genetic Disorders • Mental Retardation • Psychiatry • TSC1 • TSC2

Implementation of Etiology-Based Treatment Protocol and Early Epilepsy Monitoring Unit Follow-Up for Patients with Infantile Spasms Syndrome Improves Quality of Care and Clinical Outcomes (AES 2025) - "A prior study at our institution (study cohort from 2015-2019) found that only 46% of infants treated with adrenocorticotropic hormone (ACTH) and 54% with vigabatrin (VGB) responded to treatment... Timely electro-clinical follow-up is essential for infantile spasms. The implementation of our standardized, etiology-driven protocol—paired with EMU-based follow-up has significantly shortened the time to follow-up and improved treatment responsiveness and standardizing care at our center. Early EMU follow-up, supported by health insurers, enables extended EEG monitoring for better detection of subtle spasms, facilitates timely treatment adjustments, and accelerates surgical evaluation for medically refractory cases, ultimately resulting in improved outcomes for this time-sensitive epileptic syndrome."

Clinical • Clinical data • CNS Disorders • Epilepsy

Management of Infantile Epileptic Spasms Syndrome: A Survey of U.S. Tertiary Pediatric Hospitals (AES 2025) - "Clinical practice across PERC centers has evolved according to evolving evidence, generating broad consensus regarding the use of high-dose prednisolone and sequential therapy, increased use of the BASED score, and ubiquity of genetic testing in most cases. However, there were more discrepancies regarding second and third-lines therapies, duration of vigabatrin therapy in responders, and criteria for referral for epilepsy surgery. These findings underscore the need for updated evidence-based guidelines to standardize care for IESS nationwide."

Clinical • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • Pediatrics • Psychiatry

Predictors of Relapse of Infantile Epileptic Spasms Syndrome After Initial Response to Vigabatrin (AES 2025) - "These data suggest—but far from confirm—that continued vigabatrin (100 to 149 mg/kg/day) may protect against IESS relapse. Vigabatrin—or any potential relapse prevention strategy—is most needed for patients with known etiology, other seizure-types, delayed initiation of vigabatrin, and history of prior IESS relapse. Further prospective study is needed to validate observations described in this study."

CNS Disorders • Epilepsy

Response to Anti-seizure Medications in Children Carrying Novel or Previously Reported HCN1 Gene Variants. (AES 2025) - "Levetiracetam (LEV) was the most frequently used drug, and of ASMs with at least three exposures, cannabidiol (CBD) the least used...However, clobazam (CLB) and valproate (VPA) demonstrated significantly better efficacy, with VPA the most effective of all ASMs...Two ASMs caused worsening of seizure frequency: LTG and oxcarbazepine (OXC). Other ASMs perceived as having no benefit were lacosamide (LAC), phenobarbital (PB), vigabatrin (VGB), and carbamazepine (CBZ). The one subject who was deceased due to SE was being treated at the time with LTG and topiramate (TPM). We report eight novel HCN1 variants... We report eight novel HCN1 variants. Of these, variants mapping to an ion channel transmembrane domain caused more severe phenotypes. In subjects with epilepsy, LTG and OXC exacerbated seizure frequency whereas VPA and CLB improved it, with two subjects becoming seizure-free after exposure to these ASMs."

Clinical • CNS Disorders • Developmental Disorders • Epilepsy • Psychiatry

Comparison of Effectiveness and Safety of Nitrazepam Versus Topiramate in Resistant Infantile Epileptic Spasms Syndrome: An Open-Label, Randomized Controlled Trial (AES 2025) - "Rationale: Globally, there are differences in management practices among pediatric neurologists in choosing antiseizure medicine in children with infantile epileptic spasms syndrome (IESS) after failing to hormonal and vigabatrin therapy. Our trial provides proof-of-concept of the superior short-term effectiveness of nitrazepam over topiramate therapy in resistant IESS. It also demonstrated better quality of life improvements and autonomic stability. Further studies are needed to evaluate its long-term efficacy and safety."

Clinical • Anesthesia • 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

Outcomes in West syndrome: Association of genetic and perinatal etiologies with early diagnosis and therapy. (PubMed, Pediatr Int) - "Genetic and perinatal etiologies fundamentally shape both neurodevelopmental and seizure outcomes in West syndrome. Early diagnosis, comprehensive etiological evaluation, and personalized therapy-favoring monotherapy where feasible-are critical for optimizing prognosis. These findings underscore the necessity of etiological stratification and tailored interventions to improve long-term outcomes in affected infants."

Journal • Retrospective data • Cardiovascular • CNS Disorders • Developmental Disorders • Epilepsy • DEPDC5 • TUBA1A

Infantile Spasms in Inborn Errors of Metabolism: Diagnostic and Therapeutic Considerations. (PubMed, J Child Neurol) - "Levetiracetam was started, and vigabatrin was introduced following liver transplant, which resulted in seizure freedom. The patient was later treated with two 4-week courses of enteral prednisolone, leading to the cessation of spasm. These cases emphasize the complexity in managing infantile epileptic spasms syndrome in patients at high risk for metabolic decompensation."

Journal • CNS Disorders • Epilepsy • Genetic Disorders • Metabolic Disorders • Transplantation

Case Report: Adenylosuccinate lyase deficiency type I caused by splicing disruption due to a novel missense variant in the ADSL gene. (PubMed, Front Genet) - "Seizures were temporarily controlled with ethosuximide and vigabatrin, though neurodegeneration progressed. This case highlights splicing disruption as a novel pathogenic mechanism in ALD and expands the mutational spectrum associated with the disease. This case also underscores the importance of integrating RNA analysis with genomic data to uncover cryptic splicing defects, especially when protein-level predictions suggest benignity."

Journal • CNS Disorders • Developmental Disorders • Epilepsy • Metabolic Disorders

Epidemiology of Epileptic Spasm and Affecting Factors on One-Year Prognosis: A Study in Tabriz Children's Hospital. (PubMed, Iran J Child Neurol) - "Treatment involved Phenobarbital in 35 patients (94.6%), Vigabatrin in 29 (78.4%), and ACTH in 11 (29.7%). The most common causes of ES are prenatal insults, cryptogenic factors, and CNS malformations. The key one-year prognoses include disease recurrence and recovery with complications."

Journal • Cardiovascular • CNS Disorders • Epilepsy

Neonatal ethylmalonic encephalopathy with neuroradiological lesions at birth: is it an in utero disease? (SSIEM 2023) - "Partial control of the seizures was obtained with Vigabatrin (100 mg/kg/d)... Newborn screening allows a rapid diagnosis of the disease. In our case, neuroradiological abnormalities were surprisingly already present at birth, leading to hypothesize a pre-perinatal disease onset with progressive neurological worsening despite the absence of other symptoms. Alternative therapies, such as liver transplantation, may not represent an option in cases with predominant neurological presentation."

CNS Disorders • Dystonia • Epilepsy • Gene Therapies • Metabolic Disorders • Perinatal Disorders