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.

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.

Clinical Trial Conditions for vigabatrin

Condition Name

Condition Name for vigabatrin
Intervention	Trials
Cocaine Dependence	5
Infantile Spasms	5
Infantile Spasm	4

Condition MeSH

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

Clinical Trial Locations for vigabatrin

Trials by Country

Trials by Country for vigabatrin
Location	Trials
United States	96
Poland	2
France	2
Korea, Republic of	1
Spain	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

Last updated: July 27, 2026

Vigabatrin clinical trials update, market analysis, and 2030+ projection

Vigabatrin is an established antiseizure therapy used for infantile spasms (West syndrome) and refractory focal seizures with impaired awareness (FS-IA). Its clinical development pipeline is comparatively narrow versus newer antiseizure drugs, with most activity focused on optimizing use, monitoring, and assessing long-term safety (especially ocular toxicity). Commercial outlook is driven by (1) persistent specialty prescribing in rare pediatric epilepsy indications, (2) regulatory surveillance requirements, and (3) constrained generic entry where market access depends on state-level prescribing controls and payer coverage.

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What is vigabatrin approved for and what is its FDA status?

Core FDA indications (U.S.)
Vigabatrin is approved for:

• Infantile spasms (West syndrome) in patients ≥ 1 month.
• Refractory complex partial seizures (historically) / focal seizures with impaired awareness (current label terminology varies by reference) in patients who have failed at least two alternative therapies, where benefit outweighs the risk.

Regulatory monitoring and risk management
The defining regulatory feature for vigabatrin is its ocular toxicity risk. U.S. labeling includes requirements for:

• Baseline ophthalmologic assessment
• Ongoing vision monitoring (frequency specified in labeling)
• Patient counseling and documentation supporting benefit-risk management.

Impact on market
These requirements raise administrative friction and payer scrutiny, limiting addressable volumes relative to drugs without similar monitoring mandates.

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What clinical trials have reported results for vigabatrin in the last 5–10 years?

Recent update theme: real-world risk-benefit optimization and long-term follow-up
Across vigabatrin’s later-period evidence base, the dominant “trial” activity has been:

• Long-term observational follow-up and registry-like studies capturing retinal toxicity incidence and treatment discontinuation patterns.
• Studies evaluating monitoring adherence, including visual field testing performance and barriers.
• Pediatric subgroup analyses relevant to infantile spasms treatment pathways.

Pipeline signal quality
Vigabatrin’s pipeline is not characterized by large phase 3 programs typical of late-stage antiseizure candidates. The evidence base largely reflects:

• Early pivotal trials that established efficacy (infantile spasms and refractory focal seizures), and
• Post-approval safety characterization and risk management.

Where trial activity typically clusters

• Neurodevelopmental outcomes in infantile spasms treated with vigabatrin-containing regimens.
• Ocular safety assessment methods (practicality of visual field testing in children, test reliability, and timing strategies).

Featured snippet answer
Vigabatrin’s modern clinical update is mainly safety surveillance and monitoring optimization, not major new efficacy phase 3 expansions.

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What phase 1, phase 2, or phase 3 trials exist for vigabatrin today?

What to expect structurally

• Small interventional studies, often safety, pharmacovigilance, or monitoring methodology
• Treatment pathway studies in infantile spasms and refractory focal seizures
• Studies that support guideline positioning rather than new-label expansion

Practical implication Even when trials initiate, the most likely endpoints support:

• Ocular risk mitigation practices
• Adherence feasibility
• Long-term tolerability

This structure keeps the probability of label-expanding outcomes lower than for novel antiseizure agents with new mechanisms or delivery systems.

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How do ocular toxicity and monitoring requirements affect clinical trial design and adoption?

Ocular toxicity as the main constraint
Vigabatrin carries a risk of retinal dysfunction and associated visual field defects. This drives:

• Strong selection criteria in trials
• Prespecified ophthalmology endpoints and monitoring schedules
• Frequent discontinuation considerations in long-term use

Effect on enrollment and retention

• Pediatric trials face additional burdens because visual field testing can be challenging in very young patients.
• Trial designs emphasize measurable ophthalmologic outcomes, which can reduce enrollment scalability.

Adoption impact Clinicians and institutions increasingly weigh vigabatrin against alternative therapies with:

• Lower long-term ocular risk profiles
• Less burdensome monitoring requirements

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How does vigabatrin perform versus other therapies for infantile spasms?

Competitive set by use case For infantile spasms, standard competitive options often include:

• ACTH-based regimens
• Steroid therapies
• Other antiseizure drugs used off-label or in-adjacent labeling depending on jurisdiction
• Emerging therapies in the pediatric epilepsy landscape

Commercial implication Vigabatrin remains a specialty option when:

• First-line therapies fail or are contraindicated
• Patient-specific risk-benefit favors vigabatrin under strict monitoring

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How does vigabatrin compare versus other options for focal seizures with impaired awareness?

Competitive set Refractory focal seizures face competition from newer antiseizure drugs with:

• Broader formularies
• No requirement for long-term visual field monitoring
• Better tolerability narratives

Market positioning Vigabatrin’s niche is typically:

• Multi-therapy failure
• Cases where clinicians accept ocular risk as the tradeoff for seizure control

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What is the current market size and sales trajectory for vigabatrin?

Market characteristics

• Low volume, high monitoring cost: use is concentrated in rare epilepsy populations and specialty clinics.
• Payer controls: coverage is influenced by documented therapeutic failure and monitoring compliance.
• State and institutional protocols: ocular monitoring operationalizes “access.”

Revenue drivers

• Persistence in the treated population (infantile spasms cohorts have episodic onset, but many patients need urgent seizure stabilization)
• Ongoing use duration for refractory focal seizures (variable by response and tolerance)
• Institutional comfort level with the drug’s risk profile and testing capacity

Revenue headwinds

• Clinician preference shifts to newer drugs with fewer monitoring burdens
• Potential drop-off in prescribing where ophthalmology access or testing capability is limited

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What is the demand outlook for infantile spasms and refractory focal seizures where vigabatrin is used?

Demand profile

• Infantile spasms incidence is low; demand is largely linked to early referral and treatment urgency.
• Refractory focal seizures are larger in absolute terms but the subset eligible for vigabatrin is smaller due to prior therapy requirements and risk constraints.

Structural forecast drivers

• Pediatric neurology treatment guidelines and pathways
• Growth in diagnostic intensity (earlier detection) raises treated counts, but does not guarantee vigabatrin share growth.
• Generic and payer dynamics can shift share only if access barriers are reduced. For vigabatrin, monitoring and risk management often remain the limiting factor even when supply economics improve.

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What 2030+ market projection is most likely for vigabatrin?

Base-case view A realistic forecast is stable to modestly declining U.S. share, with the drug retaining a role in refractory or second-line settings under strict monitoring. Market growth, if any, is more likely to come from:

• Increased diagnosis and earlier intervention in pediatric epilepsy
• Sustained use in refractory cases

Key reason share growth is hard Vigabatrin’s ocular toxicity profile acts like a durable competitive disadvantage against newer antiseizure therapies and alternative pathways for infantile spasms.

Projection shape

• Near term (1–3 years): stable revenue with volatility tied to monitoring compliance and payer coverage.
• Mid term (4–7 years): gradual share erosion if prescriber preference shifts continue.
• Long term (7–10+ years): value retention in a niche; overall category growth does not necessarily translate to vigabatrin growth.

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What factors determine the risk of generic entry for vigabatrin and what generic launch scenarios exist?

Generic entry determinants For antiseizure drugs with meaningful safety risk, competitive entry depends on:

• Patent landscape around active ingredient, formulations, and method-of-use
• Regulatory exclusivity status of the reference product and any listed patents
• Settlements that can delay entry
• Practical ability to meet risk management and monitoring requirements (if tied to prescribing controls)

Generic scenario taxonomy

• Scenario A: early entry when the reference’s listed patent estate is limited or successfully cleared.
• Scenario B: delayed entry due to patent infringement litigation or Orange Book exclusivity.
• Scenario C: partial market impact even with generic availability because monitoring requirements constrain uptake.

Featured snippet answer
Generic entry risk exists primarily through patent expiration and Orange Book clearance. Even if a generic launches, vigabatrin uptake is constrained by ocular monitoring burden and payer controls.

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What patents protect vigabatrin and how long is the remaining exclusivity?

A patent-centered answer requires current Orange Book and litigation records for the specific FDA product reference(s) (including all listed patents: active ingredient, formulations, and method-of-use). This response cannot be produced accurately without those records.

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What patent litigation affects vigabatrin in the U.S.?

An accurate litigation update also requires current dockets and Orange Book Paragraph IV records for the reference product(s). This response cannot be produced accurately without those records.

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What is the Orange Book status of vigabatrin and are there listed patents?

Orange Book status is product-specific and time-sensitive. This response cannot be produced accurately without the current Orange Book listing details for vigabatrin reference products.

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What delivery forms and formulation innovations exist for vigabatrin?

Vigabatrin is used in oral formulations (tablets and/or powder formulations depending on market availability). The commercial thesis is driven more by:

• dosing convenience,
• ability to ensure consistent exposure in pediatrics, and
• risk monitoring infrastructure

than by breakthrough formulation innovation.

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Which companies market vigabatrin and how do their commercial strategies differ?

Company-specific market positioning depends on current U.S. distribution, REMS-like operational workflows (where applicable), and payer contracts. This response cannot be produced accurately without up-to-date company-level sales data and current labelholder/manufacturer information.

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How strong is the competitive landscape for vigabatrin in epilepsy?

Competitors by therapeutic niche

• Newer antiseizure drugs in focal epilepsies often capture share due to simpler long-term safety narratives and broader payer acceptance.
• For infantile spasms, ACTH and steroid pathways and other pediatric treatments can reduce reliance on vigabatrin in first-line or second-line use.

Structural competitive disadvantage

• Persistent ocular risk requiring long-term monitoring
• Higher cost of care beyond the drug price

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Key Takeaways

• Vigabatrin remains a niche antiseizure therapy centered on infantile spasms and refractory focal seizures with strict ocular safety monitoring.
• Clinical updates in recent years emphasize safety surveillance, ophthalmologic monitoring practices, and long-term outcomes, not major efficacy label expansion.
• Market trajectory is most consistent with stable-to-modest decline in share as clinicians favor antiseizure alternatives with less burdensome risk management.
• Future growth, if any, depends on diagnostic intensity and continued specialist use in refractory cases, not on broad adoption.

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FAQs

1. Does vigabatrin have REMS in the U.S. and what monitoring does it require?
2. How often are ophthalmologic tests recommended for patients on vigabatrin?
3. What alternatives are used when infantile spasms do not respond to first-line therapy?
4. How does payer coverage usually handle vigabatrin for refractory focal seizures?
5. Are there significant regional differences in vigabatrin availability and prescribing restrictions?

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References

1. FDA. Prescribing Information for vigabatrin (U.S. label). U.S. Food and Drug Administration.
2. ClinicalTrials.gov. Search results for vigabatrin clinical trials. National Library of Medicine.