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.
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 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.
NCT01073579 ↗	Sabril Patient Registry	Completed	Lundbeck LLC		2009-08-01	The purpose of this study is to create a patient registry to collect and analyze information on subjects treated with Sabril and the prescribers of Sabril.
NCT01266291 ↗	Sabril for Complex Partial Seizures in Adult Tolerability Study (TS) Patients	Terminated	H. Lundbeck A/S	Phase 4	2010-08-01	This is an open-label, phase 4 study to examine the safety and efficacy of vigabatrin (Sabril) in Tuberous Sclerosis patients, a subset of the larger refractory complex partial epilepsy population for which the drug is approved. While enrolled on this trial, subjects will continue to take all of their normally prescribed medications, including their other antiepileptic drugs (AEDs). Alternatively, there is a prospective observational arm that subjects who are about to take Sabril as treatment for seizures associated with Tuberous Sclerosis may join. Subjects who join this arm will not have any study visits and will not be asked to do anything specifically for the study. The study team will collect all study data from subjects' medical records only.

Clinical Trial Conditions for SABRIL

Condition Name

Condition Name for SABRIL
Intervention	Trials
Infantile Spasms	3
Cocaine Dependence	2
Infantile Spasm	2
Therapeutic Equivalency	1

Condition MeSH

Condition MeSH for SABRIL
Intervention	Trials
Spasms, Infantile	5
Spasm	5
Seizures	3
Muscle Cramp	2

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
Oregon	2
North Carolina	2
Minnesota	2
Florida	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
Active, not recruiting	1

Clinical Trial Sponsors for SABRIL

Sponsor Name

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

Sponsor Type

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

Sabril (vigabatrin): Clinical Trials Update, Market Analysis, and Projection

Last updated: April 28, 2026

What is Sabril and what is its regulatory status profile?

Sabril is vigabatrin, an antiepileptic drug used for refractory seizures. In practice, the commercial footprint is shaped by the drug’s special safety monitoring requirements, limited approved indications by region, and long-cycle prescribing tied to specialized epilepsy care.

Key market-impacting attribute

• Safety program constraints: Vigabatrin is associated with risk of irreversible vision loss, which has driven risk mitigation programs that restrict routine prescribing and can slow patient starts. (The restriction dynamic affects near- and medium-term demand stability versus broader-spectrum antiepileptics.)

What does the clinical trials pipeline look like for Sabril now?

Sabril’s clinical activity is not characterized by a broad modern phase-3 regimen. The drug is marketed as an established therapy, and trial effort is largely expected to concentrate on:

• New formulations, dosing strategies, or delivery to improve tolerability and adherence
• Observational outcomes tied to safety monitoring, real-world effectiveness, and long-term visual risk management
• Regulatory updates tied to region-specific label and safety program changes

Net implication for investors and R&D decision-makers

• Sabril’s growth does not depend on a traditional late-stage pipeline re-rating.
• Forecast sensitivity shifts to label scope changes, safety program evolution, payer coverage, and competitive dynamics rather than to breakthrough efficacy readouts.

How big is the Sabril market today and what drives demand?

Sabril competes in a narrow slice of epilepsy treatment where clinicians use vigabatrin when other options fail or when specific patient profiles align with label indications.

Demand drivers

1. Treatment positioning
   • Refractory epilepsy usage patterns concentrate prescribing in specialized centers.
2. Safety monitoring capacity
   • The vision-risk management burden affects initiation rates and retention.
3. Substitution pressure
   • Newer antiseizure medicines (ASMs) across major markets can pull prescribers away, especially when comparative efficacy in refractory populations is favorable and when monitoring burden is lower.
4. Payer behavior
   • Coverage decisions typically depend on documentation of failure of other therapies and on ongoing compliance with safety protocols.

Market structure

• Geography: Sales are typically concentrated in countries with stable reimbursement and a mature specialized epilepsy network.
• Channel: Neurology and hospital specialty channels dominate; long-tail demand depends on ongoing patient retention.

What are the competitive dynamics for vigabatrin?

Vigabatrin faces competition from:

• Newer ASMs that can be started earlier in refractory pathways
• Treatment algorithms that increasingly favor agents with lower monitoring friction
• In some regions, alternative routes to access vigabatrin that rely on center-level protocols

Practical result: competition tends to compress net new patient starts even when some continued demand persists from existing treated patients.

What is the market projection path for Sabril (2026-2030)?

Base case: mid-single-digit growth

Given the established status of vigabatrin, projected performance typically depends on incremental wins rather than large label expansions:

• Existing patient retention contributes a floor to sales
• Modest new starts depend on specialized center utilization and payer acceptance
• Competitive substitution caps upside

Expected pattern (directionally):

• Growth is steady, not step-change
• Volatility is driven by safety-program operational capacity and payer policy tightening or loosening

Bull case: low-to-mid single-digit acceleration

Upside scenarios rely on:

• Expanded or clarified label in one or more major markets
• Improvements in safety monitoring workflow leading to higher initiation rates
• Competitive disruptions (supply, access, or coverage setbacks for alternative therapies)

Bear case: flat-to-negative net sales

Downside scenarios occur if:

• Payer coverage tightens for refractory pathways
• Monitoring requirements increase administrative barriers
• Alternative ASMs gain share in refractory treatment algorithms

What does this mean for valuation and R&D strategy?

For R&D decisions

Sabril is not a “pipeline-dependent” franchise. The only R&D levers that can realistically move the needle are those that reduce friction:

• Safety monitoring simplification
• Better tolerability or route-of-administration improvements
• Evidence generation for outcomes that matter to payers and clinicians

For investment positioning

The market is best viewed as:

• Defensible but constrained by monitoring burden
• Sensitive to reimbursement and access rather than to trial-result re-rating

Clinical trials update: what to watch

Even without a high-visibility phase-3 cadence, decision-grade monitoring points include:

• Label and safety program updates in major jurisdictions
• Real-world outcomes publications tied to effectiveness and long-term safety monitoring execution
• Comparative studies versus newer ASMs in refractory epilepsy pathways
• Regulatory communications that affect prescriber behavior (initiation thresholds, compliance expectations)

Key Takeaways

• Sabril (vigabatrin) is an established refractory epilepsy therapy with demand shaped more by safety monitoring access and reimbursement than by new late-stage clinical catalysts.
• Clinical trial activity is likely limited in scale and focused on safety, monitoring practices, and incremental optimization rather than disruptive efficacy readouts.
• Market projection for 2026 to 2030 is steady-to-moderate under most scenarios, with upside requiring label or access improvements and downside driven by tighter coverage or substitution by newer ASMs.
• The franchise should be evaluated as a specialty, constrained-growth product where operational and payer factors drive net sales more than pipeline surprises.

FAQs

1. Is Sabril’s growth dependent on new phase-3 results?
   No. The franchise is shaped primarily by safety program execution, label limits, and prescribing access.

2. What most affects new patient starts for vigabatrin?
   Safety monitoring capacity and payer requirements for refractory treatment documentation.

3. How do newer antiseizure medicines impact Sabril?
   They can reduce initiation rates by shifting treatment algorithms toward therapies with lower monitoring burden.

4. What would create upside for Sabril between 2026 and 2030?
   Label expansion or safety program improvements that reduce initiation friction and increase payer willingness.

5. What would most likely create downside?
   Coverage tightening, increased administrative burden around vision-risk protocols, or share loss to newer ASMs in refractory pathways.

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References (APA)

[1] U.S. Food and Drug Administration. (n.d.). Sabril (vigabatrin) prescribing information. FDA.
[2] European Medicines Agency. (n.d.). Sabril (vigabatrin) product information and risk management materials. EMA.