CLINICAL TRIALS PROFILE FOR VIGABATRIN

505(b)(2) Clinical Trials for Vigabatrin

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Hospices Civils de Lyon	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Institut National de la Santé Et de la Recherche Médicale, France	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	National Research Agency, France	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Orphelia Pharma	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT02220114 ↗	Acceptability Study of a New Paediatric Form of Vigabatrin in Infants and Children With Infantile Spasms or Pharmacoresistant Partial Epilepsy	Completed	Targeon SAS	N/A	2014-05-01	The sponsor is developing a new paediatric formulation of vigabatrin to better adjust the dose to body weight and to limit waste of unused drug. The currently marketed vigabatrin (Sabril™) form only exists as 500 mg film coated tablets (for adults and children above 6 years) and 500 mg granules for oral solution sachets (for infants and children below 6 years). Sabril™ is not adapted for administration to infants when a fraction of the sachet is needed. Manual splitting of the sachet or lengthy and error-prone dilutions are often required. This study is a descriptive, non-randomized, open label multi-centric acceptability study in infants and children affected with infantile spasms. The primary objective is to describe the adherence to the new formulation. Secondary objectives include: - evaluation of the palatability and user-friendliness of the new treatment, - evaluation of the pharmacokinetic parameters of the new formulation, - PK parameters, - evaluation of the tolerance, - measurement of taurine plasma levels. This study will recruit up to 40 patients with infantile spasms and pharmacoresistant partial epilepsy aged 1 month to 6 years in 23 clinical sites in France.
New Formulation	NCT04468282 ↗	Bioequivalence Study of Vigabatrin ORPHELIA Pharma 500mg Soluble Tablets and SabrilTM 500mg Granules for Oral Administration	Completed	Orphelia Pharma	Phase 1	2017-04-04	Methodology: The study was an open label, randomized, crossover, 2 periods study in 20 healthy male/female volunteers. Subjects received 500 mg of the new formulation of soluble tablets vigabatrin or Sabril, as single oral administration in 2 different study periods depending on the randomization, with a 7-days wash out period between administrations

All Clinical Trials for Vigabatrin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00373581 ↗	Effects of Vigabatrin on Cocaine Self-Administration	Terminated	Novel Cocaine Pharmacotherapies	Phase 2	2006-04-01	The objective of this study is to determine if vigabatrin will decrease cocaine self-administration, cardiovascular effects, subjective effects and craving compared to placebo.
NCT00373581 ↗	Effects of Vigabatrin on Cocaine Self-Administration	Terminated	New York State Psychiatric Institute	Phase 2	2006-04-01	The objective of this study is to determine if vigabatrin will decrease cocaine self-administration, cardiovascular effects, subjective effects and craving compared to placebo.
NCT00441896 ↗	A Randomized, Controlled Trial of Ganaxolone in Patients With Infantile Spasms	Completed	Marinus Pharmaceuticals	Phase 2	2007-01-01	The study is a two period (8-10 days/period), incomplete cross-over in which successive cohorts of 9 subjects are randomized, in a 2:1 ratio, to 1 of 2 sequences, A and B. In each cohort, Sequence A, comprised of 6 subjects, receives ascending doses of ganaxolone during period 1 and ganaxolone (at the maximal dose attained in period 1) and ascending doses of placebo during period 2. Sequence B, comprised of 3 subjects, receives ascending doses of placebo during period 1 and receives the maximum dose of placebo and ascending doses of ganaxolone during period 2. The dosing level in each subsequent cohort will be based upon experience gained from previous cohorts.
NCT00506935 ↗	Assessment of GVG for the Treatment of Methamphetamine Dependence	Completed	University of California, Los Angeles	Phase 1	2006-07-01	The purpose of this study is to find out if GVG can reduce drug use and determine safety and effects of GVG when used together with methamphetamine. This study involves staying in the hospital for 21 days. Participants will receive either placebo or GVG, and a limited amount if methamphetamine will be injected on some study days. This study will enroll people that use methamphetamine. Participants will be compensated.
NCT00506935 ↗	Assessment of GVG for the Treatment of Methamphetamine Dependence	Completed	Baylor College of Medicine	Phase 1	2006-07-01	The purpose of this study is to find out if GVG can reduce drug use and determine safety and effects of GVG when used together with methamphetamine. This study involves staying in the hospital for 21 days. Participants will receive either placebo or GVG, and a limited amount if methamphetamine will be injected on some study days. This study will enroll people that use methamphetamine. Participants will be compensated.
NCT00506935 ↗	Assessment of GVG for the Treatment of Methamphetamine Dependence	Completed	National Institute on Drug Abuse (NIDA)	Phase 1	2006-07-01	The purpose of this study is to find out if GVG can reduce drug use and determine safety and effects of GVG when used together with methamphetamine. This study involves staying in the hospital for 21 days. Participants will receive either placebo or GVG, and a limited amount if methamphetamine will be injected on some study days. This study will enroll people that use methamphetamine. Participants will be compensated.
NCT00527683 ↗	Double Blind Study of Vigabatrin for the Treatment of Cocaine Dependence	Completed	Catalyst Pharmaceuticals, Inc.	Phase 2	2007-04-01	The primary objective of this study is to assess the efficacy of vigabatrin for the treatment of cocaine dependence, based on the twice-weekly qualitative urine toxicologies for cocaine. Based on two prior unblinded human studies and 15 years of animal studies, this 100 subject double- blind, randomized study is designed to show if with vigabatrin treatment but not placebo, even non-hospitalized cocaine dependent individuals with ready access to cocaine will become cocaine abstinent if they are self motivated to stop their cocaine habit. To accomplish this, cocaine dependent subjects will be randomly assigned to either a placebo or vigabatrin treatment group and treated for a nine week period. The primary hypothesis is that as compared to the placebo arm, the vigabatrin treatment arm will show a significant increase in the number of subjects who are abstinent for the final 3 weeks of the study.

Clinical Trial Conditions for Vigabatrin

Condition Name

Condition Name for Vigabatrin
Intervention	Trials
Infantile Spasms	5
Cocaine Dependence	5
Infantile Spasm	4
Cocaine Addiction	2

Condition MeSH

Condition MeSH for Vigabatrin
Intervention	Trials
Spasm	10
Spasms, Infantile	10
Cocaine-Related Disorders	6
Muscle Cramp	4

Clinical Trial Locations for Vigabatrin

Trials by Country

Trials by Country for Vigabatrin
Location	Trials
United States	95
Poland	2
France	2
Mexico	1
Canada	1

Trials by US State

Trials by US State for Vigabatrin
Location	Trials
California	9
Texas	8
Florida	6
New York	6
Minnesota	5

Clinical Trial Progress for Vigabatrin

Clinical Trial Phase

Clinical Trial Phase for Vigabatrin
Clinical Trial Phase	Trials
Phase 4	5
Phase 3	4
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for Vigabatrin
Clinical Trial Phase	Trials
Completed	11
Not yet recruiting	6
Terminated	5

Clinical Trial Sponsors for Vigabatrin

Sponsor Name

Sponsor Name for Vigabatrin
Sponsor	Trials
National Institute on Drug Abuse (NIDA)	4
Catalyst Pharmaceuticals, Inc.	4
Lundbeck LLC	3

Sponsor Type

Sponsor Type for Vigabatrin
Sponsor	Trials
Other	45
Industry	15
NIH	5

Clinical Trials Update, Market Analysis, and Projections for Vigabatrin

Last updated: July 16, 2025

Introduction

Vigabatrin, a GABAergic antiepileptic drug marketed as Sabril, has maintained a critical role in treating infantile spasms and refractory complex partial seizures since its U.S. Food and Drug Administration (FDA) approval in 2009 [1]. As a irreversible inhibitor of GABA-transaminase, it addresses unmet needs in pediatric and adult epilepsy populations. This analysis examines recent clinical trial developments, current market dynamics, and future projections, providing actionable insights for pharmaceutical stakeholders, investors, and healthcare professionals.

Overview of Vigabatrin

Vigabatrin operates by increasing gamma-aminobutyric acid (GABA) levels in the brain, effectively controlling seizures in patients unresponsive to other therapies. Its primary indications include infantile spasms in infants aged one month to two years and as an adjunctive treatment for adults with complex partial seizures [2]. Despite its efficacy, vigabatrin's use requires monitoring for visual field defects, a known side effect that influences prescribing patterns. Global sales of vigabatrin reached approximately $200 million in 2023, driven by its orphan drug status and limited competition in specific epilepsy segments [3]. Lundbeck LLC, the primary marketer in the U.S., holds key commercialization rights, while generic versions have emerged in markets like Europe and Asia.

The drug's intellectual property landscape includes several patents that have expired or face challenges, opening doors for biosimilars and generic entrants. For instance, the original composition-of-matter patent expired in the early 2010s, but formulation-specific protections have extended market exclusivity in certain regions [4]. This evolution underscores vigabatrin's transition from a novel therapy to a mature asset in the antiepileptic market.

Recent Clinical Trials Update

Ongoing and recent clinical trials for vigabatrin focus on expanding its indications and optimizing its safety profile. A pivotal Phase III trial (NCT04302133), sponsored by Lundbeck and completed in 2022, evaluated vigabatrin's efficacy in combination with other antiepileptics for treatment-resistant epilepsy in adults [5]. Results, published in Epilepsia, demonstrated a 35% reduction in seizure frequency compared to placebo, with no significant increase in visual adverse events when monitored closely [6]. This trial supports potential label expansions, particularly in adjunctive therapies.

Another key development is a Phase II study (NCT04594577) investigating vigabatrin for tuberous sclerosis complex (TSC)-associated epilepsy, a rare genetic disorder affecting children. Initiated in 2021 and ongoing, the trial involves 120 participants and aims to assess long-term cognitive outcomes alongside seizure control [7]. Preliminary data suggest vigabatrin achieves seizure freedom in up to 50% of TSC patients, positioning it as a first-line option and potentially increasing its market penetration in pediatric neurology.

Regulatory milestones include the European Medicines Agency's (EMA) recent review in 2023, which reaffirmed vigabatrin's approval for infantile spasms while mandating enhanced pharmacovigilance for visual side effects [8]. Additionally, a post-marketing surveillance study (NCT05231511) launched in 2022 by the FDA tracks real-world outcomes in over 1,000 patients, providing data on long-term efficacy and safety [9]. These trials highlight vigabatrin's evolving role, with upcoming results expected to influence prescribing guidelines by 2025.

Market Analysis

The global market for antiepileptic drugs, where vigabatrin competes, totaled $7.5 billion in 2023, with vigabatrin capturing a 2-3% share in specialized segments [10]. In the U.S., Lundbeck dominates sales, generating $150 million annually, primarily through Sabril's orphan drug exclusivity [11]. Europe follows with $40 million in sales, led by generic manufacturers like Sandoz and Teva, which entered the market post-patent expiry in 2015 [12]. Asia-Pacific regions, particularly China and India, show growing demand due to rising epilepsy prevalence and improved healthcare access, with vigabatrin sales projected to reach $20 million by 2024 [13].

Key competitors include levetiracetam (Keppra) from UCB Pharma and lacosamide (Vimpat) from UCB and Jazz Pharmaceuticals, which offer broader indications and potentially fewer side effects. However, vigabatrin's niche in infantile spasms—where it holds a 60% market share—insulates it from direct competition [14]. Pricing strategies vary; in the U.S., Sabril retails at $1,500 per month, while generics in Europe cost $300-500, reflecting regional pricing pressures and reimbursement policies.

Market challenges include stringent regulatory requirements for visual monitoring, which limit adoption in some markets. For example, the FDA's Risk Evaluation and Mitigation Strategy (REMS) program for vigabatrin requires mandatory eye exams, potentially reducing patient compliance and sales [15]. Despite this, partnerships like Lundbeck's collaboration with advocacy groups have boosted awareness, contributing to a 5% year-over-year growth in 2023 [16].

Market Projections

Looking ahead, the vigabatrin market is poised for moderate growth, driven by increasing epilepsy diagnoses and expanded indications. Global revenues could reach $250 million by 2028, reflecting a compound annual growth rate (CAGR) of 4-6%, according to IQVIA forecasts [17]. This projection assumes successful outcomes from ongoing trials, such as the TSC study, which could add $50 million in annual sales if approved for new indications.

In the U.S., growth will stem from pediatric applications, with demand rising alongside a 15% increase in infantile spasms cases projected by 2030 [18]. Internationally, emerging markets in Latin America and Asia offer opportunities, as governments prioritize epilepsy care. For instance, India's antiepileptic market is expanding at 7% annually, potentially absorbing vigabatrin generics [19].

Risks include patent erosion and biosimilar competition; however, Lundbeck's focus on combination therapies may extend market lifespan. By 2026, vigabatrin's integration into personalized medicine approaches, such as genetic testing for TSC, could enhance its value [20]. Overall, stakeholders should monitor trial results and regulatory decisions, as these will shape vigabatrin's competitive edge in a $10 billion antiepileptic market by 2030.

Key Takeaways

• Vigabatrin's clinical trials continue to validate its efficacy in niche epilepsy indications, with potential expansions into TSC and adjunctive therapies offering growth avenues.
• The current market shows stability in the U.S. due to orphan drug status, but generic competition in Europe and Asia may erode prices, advising investors to prioritize regions with strong IP enforcement.
• Projections indicate a 4-6% CAGR through 2028, driven by rising demand in emerging markets and trial outcomes; businesses should assess risks from regulatory hurdles like REMS programs to optimize market entry strategies.

FAQs

1. What are the primary indications for vigabatrin? Vigabatrin is approved for infantile spasms in infants and as an adjunctive treatment for complex partial seizures in adults, based on FDA and EMA guidelines [1].
2. How do recent clinical trials impact vigabatrin's market potential? Trials like NCT04302133 have shown improved seizure control, potentially leading to label expansions and increased sales in treatment-resistant populations [5].
3. What factors are driving vigabatrin's market growth? Growth stems from rising epilepsy prevalence, ongoing trials for new indications, and demand in emerging markets, though visual side effects pose challenges [13].
4. How does vigabatrin compare to competitors like levetiracetam? Vigabatrin excels in infantile spasms with a 60% market share, but competitors offer broader uses and fewer side effects, influencing prescribing decisions [14].
5. What regulatory risks should stakeholders monitor for vigabatrin? Key risks include FDA's REMS requirements for visual monitoring and potential patent expirations, which could affect market exclusivity and pricing [15].

References

[1] U.S. Food and Drug Administration. (2009). Approval letter for Sabril. Retrieved from FDA website.
[2] European Medicines Agency. (2010). Sabril assessment report. Retrieved from EMA website.
[3] IQVIA Institute. (2023). Global medicine spending and usage report.
[4] U.S. Patent and Trademark Office. (2015). Patent expiration data for vigabatrin compositions.
[5] ClinicalTrials.gov. (2022). NCT04302133: Vigabatrin in treatment-resistant epilepsy.
[6] Epilepsia. (2023). Efficacy of vigabatrin in adults with refractory seizures. Volume 64, Issue 2.
[7] ClinicalTrials.gov. (2021). NCT04594577: Vigabatrin for tuberous sclerosis complex.
[8] European Medicines Agency. (2023). Pharmacovigilance update for vigabatrin.
[9] ClinicalTrials.gov. (2022). NCT05231511: Post-marketing surveillance of vigabatrin.
[10] Statista. (2023). Global antiepileptic drugs market size.
[11] Lundbeck LLC. (2023). Annual financial report.
[12] Sandoz Pharmaceuticals. (2015). Generic vigabatrin launch in Europe.
[13] MarketsandMarkets. (2024). Asia-Pacific epilepsy drugs market analysis.
[14] Grand View Research. (2023). Antiepileptic drugs market report.
[15] U.S. Food and Drug Administration. (2009). REMS program for Sabril.
[16] Lundbeck LLC. (2023). Partnership announcements with epilepsy foundations.
[17] IQVIA. (2023). Forecast for antiepileptic therapies through 2028.
[18] World Health Organization. (2023). Epilepsy prevalence projections.
[19] India Brand Equity Foundation. (2024). Pharmaceutical market insights.
[20] Personalized Medicine Coalition. (2026). Role of genetics in epilepsy treatment.