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
Cocaine Dependence	5
Infantile Spasms	5
Infantile Spasm	4
West Syndrome	2

Condition MeSH

Condition MeSH for vigabatrin
Intervention	Trials
Spasms, Infantile	12
Spasm	10
Cocaine-Related Disorders	6
Epilepsy	5

Clinical Trial Locations for vigabatrin

Trials by Country

Trials by Country for vigabatrin
Location	Trials
United States	96
France	2
Poland	2
Thailand	1
India	1

Trials by US State

Trials by US State for vigabatrin
Location	Trials
California	9
Texas	8
Florida	6
New York	6
Pennsylvania	5

Clinical Trial Progress for vigabatrin

Clinical Trial Phase

Clinical Trial Phase for vigabatrin
Clinical Trial Phase	Trials
PHASE2	1
PHASE1	1
Phase 4	5

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	47
Industry	15
NIH	5

Clinical Trials Update, Market Analysis, and Projection for Vigabatrin

Last updated: October 28, 2025

Introduction

Vigabatrin, marketed primarily as Sabril, is an antiepileptic drug (AED) developed by Pharmacia & Upjohn and now owned by Fresenius Medical Care. Approved by the U.S. Food and Drug Administration (FDA) in 2009 for infantile spasms and later for refractory complex partial seizures, vigabatrin has carved a niche in the epilepsy management landscape. Given the evolving regulatory environment and recent clinical developments, an in-depth review of vigabatrin’s current clinical trial landscape, market dynamics, and future projections is essential for stakeholders.

Clinical Trials Landscape for Vigabatrin

Current Clinical Trials and Significance

Vigabatrin's clinical development trajectory extends beyond its initial FDA approval, with ongoing and proposed trials focusing on expanding its therapeutic indications and improving safety profiles.

1. Indications Under Investigation

• Refractory Epilepsy and Lennox-Gastaut Syndrome: Trials examine efficacy in broad epileptic syndromes characterized by drug-resistant seizures.
• Tuberous Sclerosis Complex (TSC): Several trials explore vigabatrin's potential in TSC-associated epilepsy, leveraging its mechanism to modulate GABAergic pathways.
• West syndrome in infants: Continuing research aims to optimize dosing regimens and reduce the risk of visual field defects related to long-term use.

2. Safety and Tolerability Studies

A significant focus remains on mitigating vigabatrin's most concerning adverse effect: visual field impairment. Multiple ongoing studies seek to identify biomarkers or imaging indicators to predict at-risk populations, enhance patient monitoring, and develop safer administration protocols.

Notable Trials and Updates

• NCT02989219: An ongoing Phase IV observational study assessing long-term safety in children treated for infantile spasms.
• NCT03892045: A trial examining the neurodevelopmental outcomes in infants treated with vigabatrin versus other AEDs.
• Post-Market Surveillance Studies: Renewed emphasis in regulatory jurisdictions on real-world data to monitor visual toxicity, given its significance for patent expiration and market viability.

Regulatory and Clinical Significance

Clinical trials continue to emphasize the delicate balance between efficacy and safety, particularly in pediatric populations. Regulatory agencies have issued stricter guidelines, mandating comprehensive post-marketing surveillance to mitigate risks associated with visual field defects.

Market Analysis

Market Overview and Historical Context

Vigabatrin’s market has traditionally been niche, primarily serving patients with treatment-resistant epilepsy, notably infantile spasms, where few alternatives exist. The market has been influenced by safety concerns, regulatory restrictions, and competition from newer, targeted therapies.

• Global Market Size (2022): Estimated around USD 300 million, primarily driven by North America and Europe.
• Market Share and Key Players: Fresenius Medical Care dominates, with secondary contributions from generic manufacturers once patent exclusivity lapses.

Competitive Landscape

The epilepsy market has seen significant innovation, with drugs like cannabidiol (Epidiolex), brivaracetam, and stiripentol expanding options. Vigabatrin faces competition from these agents, especially for broader indications.

Challenges Impacting Market Growth

• Safety Profile: Visual field deficits limit use, especially in pediatric populations predisposed to long-term therapy.
• Regulatory Restrictions: Stringent post-approval requirements hinder market expansion.
• Side-Effect Management: Need for robust monitoring decreases attractiveness compared to newer AEDs with more favorable safety profiles.

Market Drivers

• Unmet Needs: For refractory infantile spasms and certain epileptic syndromes, vigabatrin remains a critical option.
• Potential in Adjunctive Therapy: Ongoing trials may reinforce its position if safety concerns are effectively managed.

Market Projection and Future Outlook

Forecast Period (2023–2030)

Based on current trends, the global vigabatrin market is expected to grow at a compound annual growth rate (CAGR) of approximately 4-5%.

Drivers of Growth

• Expansion of Indications: Pending positive trial outcomes for TSC and West syndrome could significantly broaden use cases.
• Enhanced Safety Profiles: Advances in monitoring technology and personalized medicine may reduce adverse effects, encouraging wider adoption.
• Regulatory Support: Evolving guidelines favoring orphan drugs and rare disease treatments could facilitate access.

Potential Obstacles

• Emergence of New Therapies: Short-term competition from gene therapies and novel oral AEDs may temper growth.
• Reimbursement and Pricing Pressures: Cost containment measures in developed markets could impact profitability and market penetration.
• Safety-related Restrictions: Persistent safety concerns could limit use, especially for long-term therapy.

Long-term Projections

By 2030, the market could expand to approximately USD 400–500 million, contingent upon successful clinical trial outcomes, safety improvements, and regulatory adaptations. Elite positioning may be achievable with a focus on niche indications and personalized treatment approaches.

Regulatory and Commercial Strategy

Stakeholders should prioritize safety data dissemination, foster collaborations for indication expansion, and invest in innovative monitoring tools to mitigate adverse effects. Market access strategies must address reimbursement pathways, especially in emerging markets.

Key Takeaways

• Vigabatrin remains a vital therapeutic option for refractory infantile spasms and certain epileptic syndromes, despite safety concerns.
• Clinical trials are actively exploring expanded indications, with a focus on long-term safety, neurodevelopmental outcomes, and predictive biomarkers.
• The market is characterized by high unmet needs but constrained by safety profiles, regulatory restrictions, and competitive dynamics.
• Future growth hinges on enhancing safety, regulatory support, and indication expansion. Strategic investments in monitoring and personalized medicine will be critical.
• Overall, the vigabatrin market is poised for mid-term growth, especially if ongoing trials and safety improvements yield positive outcomes.

FAQs

1. What are the primary indications for vigabatrin?
Vigabatrin is FDA-approved for infantile spasms and refractory complex partial seizures. Off-label and investigational uses include treatment in TSC and Lennox-Gastaut syndrome.

2. How significant are safety concerns related to vigabatrin?
The primary safety concern is visual field loss, which can be irreversible. This risk limits widespread use, especially in young children and long-term therapy contexts.

3. Are there ongoing clinical trials expanding vigabatrin’s indications?
Yes, multiple trials, such as those examining TSC-associated epilepsy and neurodevelopmental outcomes, aim to broaden its therapeutic scope.

4. How does the market outlook look for vigabatrin?
The market is expected to grow modestly over the next decade, driven by indication expansion and safety management improvements, reaching approximately USD 400-500 million globally by 2030.

5. What strategies can improve vigabatrin’s market position?
Enhancing safety profiles through monitoring innovations, pursuing new indications, and engaging with regulatory agencies for supportive guidelines are key strategies.

References

1. FDA. (2009). Vigabatrin (Sabril) Approved for Infantile Spasms. U.S. Food and Drug Administration.
2. MarketWatch. (2022). Global Epilepsy Drug Market Size & Trends.
3. ClinicalTrials.gov. Database of ongoing vigabatrin studies.
4. European Medicines Agency. (2021). Vigabatrin safety guidelines and post-marketing surveillance.
5. Frost & Sullivan. (2023). Epilepsy Therapeutics Market Analysis and Forecasts.

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This comprehensive analysis provides business professionals with strategic insights necessary for navigating vigabatrin’s clinical and market environment.