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.

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.

Clinical Trial Conditions for VIGABATRIN

Condition Name

Condition Name for VIGABATRIN
Intervention	Trials
Cocaine Dependence	5
Infantile Spasms	5
Infantile Spasm	4
Cocaine Addiction	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
Poland	2
France	2
United Kingdom	1
Korea, Republic of	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

VIGABATRIN Market Analysis and Financial Projection

Last updated: April 28, 2026

Vigabatrin Clinical Trials Update and Market Projection

What is the current clinical-trial and regulatory status of vigabatrin?

Vigabatrin (brand: Vigabatrin/Vigadrone and in some markets Sabril) is an established anti-seizure therapy built on irreversible inhibition of GABA transaminase. Clinical development in recent years is not dominated by new late-stage efficacy trials; instead, activity centers on safety monitoring, risk management, and real-world effectiveness around long-term vision loss risk (retinal toxicity).

Key points that define the modern trial and regulatory posture

• Risk-management focus: regulatory frameworks emphasize structured ophthalmic surveillance due to permanent visual field defects that can be difficult to detect without scheduled testing.
• Trial activity pattern: new studies tend to be observational, registry-based, or geared toward optimizing monitoring protocols and patient stratification rather than running broad new Phase 3 programs.

Labeling and safety anchor (evidence basis for monitoring)

• The FDA prescribing information for Sabril includes the established warning that vigabatrin can cause irreversible vision loss, prompting requirements for ophthalmic evaluation before and during treatment (FDA label). [1]

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What does the clinical-trial landscape look like (by program type and endpoints)?

Because vigabatrin is off-patent in most major markets and sits within mature clinical use, the current “update” is best captured by the types of studies and the endpoints they target:

1) Ophthalmic monitoring and detection strategies

• Trials and post-marketing studies evaluate ophthalmic testing frequency, patient adherence, test sensitivity, and how to manage patients who show early retinal changes.
• Typical endpoints: changes in visual field measures, time-to-detection, rates of abnormal screening outcomes, and patient discontinuation.

2) Comparative effectiveness and utilization

• Studies compare outcomes in real-world settings against alternative add-on regimens for refractory epilepsies.
• Typical endpoints: seizure reduction, responder rates, adherence to monitoring, and persistence on therapy.

3) Population-specific evidence (pediatric and refractory epilepsy subtypes)

• Development priorities historically focused on infantile spasms (West syndrome), and later on refractory focal seizures.
• Current work is often narrower and designed to refine best-use recommendations rather than prove new efficacy indications.

Efficacy and safety conclusions remain label-driven

• The core efficacy claims for vigabatrin are entrenched in labeling and supported by earlier pivotal trials; recent research emphasis remains on risk mitigation and monitoring outcomes (FDA label; EMA framework summaries). [1][2]

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How is vigabatrin positioned in the epilepsy treatment market?

Vigabatrin is used in high-need refractory populations where seizure control is difficult and where clinicians accept the trade-off under mandated eye-safety protocols.

Indication footprint

• Infantile spasms (West syndrome): historically the most commercially important label segment in many markets.
• Refractory complex partial seizures (focal seizures): used as add-on when other options fail.

(Indications are consistent with major regulatory labels for vigabatrin products.) [1]

Competitive environment

The commercial threat is not direct “same mechanism” competition at scale; it is mainly:

• Second-generation anti-seizure drugs with better tolerability profiles and less burdensome long-term monitoring.
• Newer epilepsy therapies that compete for the same refractory patient share even when seizure types partially overlap.

The counterbalance for vigabatrin is:

• Strong efficacy in specific refractory seizure contexts
• Clinician familiarity and established clinical pathways for its use and monitoring

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What is the market size and where does demand come from?

A precise “current-year” global market number requires paywalled commercial datasets; however, the demand drivers are well-defined and stable:

Demand drivers

1. Refractory epilepsy incidence and duration: patients remain on therapy for long periods if effective.
2. Monitoring infrastructure: once protocols exist in a country, conversion to continued use is higher.
3. Payer coverage patterns: many jurisdictions treat vigabatrin as a specialty drug with prior authorization and monitoring compliance requirements.

Geographic reality

• Higher utilization in geographies with structured ophthalmic programs for mandated surveillance.
• Constrained usage where ophthalmology access is limited or where monitoring compliance drives discontinuation.

This pattern is consistent with the safety-driven risk management architecture embedded in product labeling (FDA label). [1]

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How should investors and planners think about near-term growth vs. structural headwinds?

Base case growth logic (2012-2020s pattern continuing)

• Uptake depends on treatment need and continued availability through generic erosion and brand access.
• Growth is tempered by monitoring burden and the emergence of alternative add-ons.

Structural headwinds

• Long-term safety concerns drive:
  • clinician caution
  • patient and caregiver hesitancy
  • earlier discontinuations after abnormal ophthalmic screening
• Competitive substitution:
  • newer anti-seizure therapies attract refractory patients when they offer similar efficacy with more favorable monitoring

Net effect expectation

• Vigabatrin market performance is more likely to track maintenance demand rather than step-change expansion, with volatility driven by generic availability and local monitoring enforcement.

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Market projection: scenarios (5-year horizon)

Without paid market-forecast datasets, the only defensible projection format is scenario-based and mechanism-linked to known constraints: safety monitoring, access, and competition. The table below models directionally how the market can move by combining (a) demand stability in refractory epilepsy, (b) generic price pressure, and (c) replacement by newer therapies.

Global vigabatrin market projection (directional, 2024 to 2029)

Scenario	Assumptions	Market trajectory
Downside	Monitoring non-compliance in some settings increases discontinuation; substitution by newer add-ons accelerates; generic penetration increases price pressure	Low single-digit CAGR decline
Base case	Monitoring programs remain stable; refractory demand sustains conversions; generic pricing continues but not catastrophically	Flat to low single-digit CAGR
Upside	Ophthalmic compliance improves; new evidence supports optimized monitoring workflow; payer pathways stabilize for refractory populations	Low single- to mid-single-digit CAGR

What makes these scenarios credible for vigabatrin

• The drug’s ceiling is tied to a safety trade-off that is managed, not eliminated, by monitoring (FDA label). [1]
• The drug’s uptake is tied to clinical need rather than novel mechanism-driven expansion.

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What will most influence revenue and utilization?

1) Ophthalmic monitoring compliance and discontinuation rates

• These determine effective treatment duration and therefore total patient-years.

2) Pricing pressure from generics

• In mature branded markets, generic competition typically drives revenue toward volume-only growth, which is constrained by monitoring limits.

3) Payer and policy restrictions

• Prior authorization requirements and monitoring mandates can either increase administrative friction (hurting utilization) or formalize appropriate access (supporting utilization).

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Key Takeaways

• Vigabatrin’s modern clinical focus is risk management, particularly ophthalmic surveillance, rather than brand-new late-stage efficacy trials. [1]
• Market demand is steady but capacity-limited by the permanent vision-loss risk and monitoring burden embedded in labeling. [1]
• Base case expectations for 2024 to 2029 are flat to low single-digit growth, with upside requiring stronger monitoring adoption and payer access stability, and downside driven by substitution and discontinuation.

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FAQs

1) What is vigabatrin’s major safety risk that shapes clinical use?

It can cause irreversible vision loss and requires structured ophthalmic evaluation and ongoing monitoring as described in regulatory labeling. [1]

2) Are there new Phase 3 efficacy trials expected to change the label soon?

Recent activity in the vigabatrin space has skewed toward monitoring, safety surveillance, and real-world evidence, with the label foundation remaining stable. [1][2]

3) Why does market growth look limited versus newer anti-seizure drugs?

Because use is constrained by long-term retinal toxicity risk and the operational burden of compliant ophthalmic testing. [1]

4) What drives utilization in practice?

High-need refractory epilepsy where clinicians choose vigabatrin due to established efficacy and accept the monitoring requirements. [1]

5) How should forecasts treat generics?

Generics typically compress price and shift performance to volume and adherence-driven persistence; growth is therefore more likely to be low unless access and monitoring improve materially.

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References

[1] U.S. Food and Drug Administration. (n.d.). Sabril (vigabatrin) prescribing information / safety information. FDA. https://www.accessdata.fda.gov/
[2] European Medicines Agency. (n.d.). Vigabatrin (Sabril) product information and risk minimisation measures. EMA. https://www.ema.europa.eu/