CLINICAL TRIALS PROFILE FOR SABRIL

505(b)(2) Clinical Trials for Sabril

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 Sabril

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
NCT00527683 ↗	Double Blind Study of Vigabatrin for the Treatment of Cocaine Dependence	Completed	New York University School of Medicine	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.
NCT00527683 ↗	Double Blind Study of Vigabatrin for the Treatment of Cocaine Dependence	Completed	NYU Langone Health	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 Sabril

Condition Name

Condition Name for Sabril
Intervention	Trials
Infantile Spasms	3
Cocaine Dependence	2
Infantile Spasm	2

Condition MeSH

Condition MeSH for Sabril
Intervention	Trials
Spasms, Infantile	5
Spasm	5
Seizures	3

Clinical Trial Locations for Sabril

Trials by Country

Trials by Country for Sabril
Location	Trials
United States	37
France	2
Thailand	1
Mexico	1
Canada	1

Trials by US State

Trials by US State for Sabril
Location	Trials
Pennsylvania	4
Texas	2
Oregon	2
North Carolina	2
Minnesota	2

Clinical Trial Progress for Sabril

Clinical Trial Phase

Clinical Trial Phase for Sabril
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	2
Phase 2	4

Clinical Trial Status

Clinical Trial Status for Sabril
Clinical Trial Phase	Trials
Completed	5
Terminated	3
Recruiting	1

Clinical Trial Sponsors for Sabril

Sponsor Name

Sponsor Name for Sabril
Sponsor	Trials
University of Pennsylvania	2
Orphelia Pharma	2
Lundbeck LLC	2

Sponsor Type

Sponsor Type for Sabril
Sponsor	Trials
Other	11
Industry	9
NIH	2

Sabril (Vigabatrin): Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 28, 2025

Introduction

Sabril (Vigabatrin) was originally approved in the early 2000s as an adjunctive therapy for partial seizures and later gained approval for infantile spasms. As a product of(goods) for refractory epilepsy, Sabril’s unique mechanism of action and regulatory landscape shape its ongoing development and market trajectory. This analysis provides a detailed update on its clinical trial landscape, market dynamics, and future growth projections, essential for stakeholders evaluating investment or competitive positioning.

Clinical Trial Landscape: Evolution and Current Status

Historical Context and Key Clinical Milestones

Vigabatrin’s initial approval was based on pivotal trials demonstrating efficacy in refractory partial seizures and infantile spasms. The drug’s mechanism involves irreversible inhibition of GABA transaminase, leading to increased GABA concentrations in the central nervous system. Due to risks of visual field defects, post-marketing surveillance has been paramount in clinical development and safety monitoring.

Recent and Ongoing Trials

Over the past five years, the clinical trial landscape for Vigabatrin has shifted towards:

• Safety and Tolerability Studies: Given the adverse effects, especially visual field constriction, numerous trials focus on optimizing dosing strategies to mitigate toxicity while maintaining efficacy. For instance, newer trials such as NCT04593884 evaluate dose reduction protocols in pediatric populations, aiming to balance benefits and risks.

• Expanded Indication Trials: A handful of studies are exploring Vigabatrin’s neuroprotective potential in neurodegenerative disorders like Parkinson’s disease (NCT03537728) and epilepsy comorbidities. These trials usually involve small cohorts and preliminary endpoints but suggest evolving interest in broader CNS applications.

• Pharmacovigilance and Long-term Safety Monitoring: Continuous post-marketing surveillance, including registries, has been reinforced through large observational studies like the Vigabatrin Registry, providing real-world safety data crucial for regulatory guidance.

Regulatory and Market Access Developments

The U.S. Food and Drug Administration (FDA) continues to emphasize risk management for Vigabatrin. Recent updates include the REMS (Risk Evaluation and Mitigation Strategy) enhancements, requiring healthcare providers to be certified and to inform patients of potential visual risks.

In Europe, the European Medicines Agency (EMA) maintains a cautious stance, with some countries restricting use primarily to severe infantile spasms unresponsive to other treatments. This regulatory environment influences ongoing and planned clinical trials, limiting access for broader indications.

Market Analysis

Market Size and Key Drivers

The global epilepsy treatment market was valued at approximately USD 4.8 billion in 2022, with the refractory epilepsy segment constituting a significant share due to the unmet need for effective therapies. Sabril’s market penetration remains concentrated in niche subpopulations, especially infants and patients with drug-resistant partial seizures.

The key drivers for Sabril include:

• Pediatric and Rare Disease Focus: As an approved treatment for infantile spasms, particularly in cases unresponsive to first-line therapies like ACTH or steroids, Sabril remains a critical second-line agent.
• Safety Profile and Monitoring Protocols: Strict REMS and monitoring requirements have constrained ease of use but simultaneously reinforce its positioning as a specialized therapy.
• Emerging Therapies and Competition: Novel drugs such as stiripentol and cannabidiol derivatives are entering the refractory epilepsy space, intensifying competition and compressing the market share of traditional drugs like Vigabatrin.

Competitor and Pipeline Overview

The epilepsy drug landscape features multiple agents targeting similar indications:

• Cannabidiol (Epidiolex): Approved for child-onset epilepsies, including Lennox-Gastaut and Dravet syndromes; offers a different safety profile with a growing market share.
• Stiripentol: Approved as adjunctive therapy for Dravet syndrome, which overlaps with infantile spasms’ patient demographics.
• Other GABAergic agents: Limited pipeline development exists, given Vigabatrin’s unique mechanism but regulatory safety concerns.

Market Trends & Challenges

• Safety Concerns: Visual field constriction remains the primary concern limiting widespread adoption outside specialized centers.
• Regulatory Restrictions: Variations across regions restrict access, and ongoing safety data collection could lead to label modifications or usage restrictions.
• Pricing and Reimbursement: As a niche medication, pricing strategies are influenced by regulatory compliance costs and limited payer incentives.

Forecast and Future Market Projection

Despite competitive pressures, Vigabatrin’s market is projected to experience modest growth through 2030, driven by:

• The sustained need for effective infantile spasm treatments.
• The potential expansion into neuroprotective and off-label CNS indications pending positive trial outcomes.
• Optimization of dosing protocols to mitigate toxicity, possibly extending its use.

Market analysts estimate a compound annual growth rate (CAGR) of approximately 2.5% to 3.2% over the next decade. By 2030, the global market for Vigabatrin could approach USD 200-$250 million, with growth primarily driven by pediatric neurology and rare disease therapeutics.

Key Considerations for Stakeholders

• Regulatory environment remains a barrier but also a barrier to new entrants; compliance and safety monitoring are critical.
• Clinical development emphasizing safety and expanding indications could unlock new markets.
• Collaborative strategies with specialty clinics and patient advocacy groups will bolster awareness and adherence.

Conclusion

Vigabatrin's ongoing clinical trials and evolving regulatory landscape influence its market trajectory. While safety concerns constrain widespread use, its niche position in treating infantile spasms and refractory epilepsy sustains steady demand. Future success hinges on optimizing dosing strategies, expanding indications through rigorous trials, and navigating regulatory requirements effectively—all pivotal to securing its role in the CNS therapeutics market.

Key Takeaways

• Vigabatrin remains essential for infantile spasms and refractory partial seizures, with ongoing trials exploring safety and new indications.
• Regulatory restrictions and safety concerns significantly influence its market penetration and growth prospects.
• The global market for Vigabatrin is expected to grow modestly, driven by demand in pediatric and rare disease segments.
• Innovation focusing on safety monitoring and expanded indications could unlock new revenue streams.
• Strategic partnerships and targeted clinical research are vital for maintaining relevance amid emerging therapies.

FAQs

1. What are the primary safety concerns associated with Vigabatrin?
The main concern is irreversible visual field constriction, leading to regulatory restrictions and mandatory monitoring protocols to mitigate this risk.

2. Is Vigabatrin approved for adult epilepsy beyond infantile spasms?
Yes, it is approved as an adjunctive therapy for refractory partial seizures in adults, primarily in the U.S. and some European countries, with usage influenced by safety considerations.

3. Are there ongoing efforts to expand Vigabatrin’s indications?
Multiple clinical trials are examining its potential neuroprotective roles, off-label uses, and safety optimization protocols, but regulatory approval for new indications remains pending.

4. How does Vigabatrin compare to emerging therapies like cannabidiol?
While cannabidiol offers a different safety profile and broader approval for various epileptic syndromes, Vigabatrin’s niche remains highly specific—though competition and label restrictions are increasing.

5. What strategies can enhance Vigabatrin’s market viability?
Focusing on safety management, expanding indications through clinical research, and engaging specialized treatment centers will be critical for sustaining and growing its market share.

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Sources:

[1] U.S. Food and Drug Administration (FDA). Vigabatrin REMS Program.
[2] European Medicines Agency (EMA). Sabril (Vigabatrin) Summary of Product Characteristics.
[3] Market Research Future. Global Epilepsy Drugs Market Analysis Report.
[4] ClinicalTrials.gov. Vigabatrin-related Trials.
[5] World Health Organization (WHO). Essential Medicines List.