CLINICAL TRIALS PROFILE FOR RUFINAMIDE

All Clinical Trials for RUFINAMIDE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00334958 ↗	Rufinamide Given as Adjunctive Therapy in Participants With Refractory Partial Seizures	Completed	Eisai Inc.	Phase 3	2006-02-13	To evaluate the effect of rufinamide on total partial seizure frequency in adolescent and adult participants (12 to 80 years, inclusive) with refractory partial onset seizures maintained on a maximum of 3 stable antiepileptic drugs (AEDs).
NCT00448539 ↗	Open-Label Extension Study of Rufinamide Given as Adjunctive Therapy in Patients With Refractory Partial Seizures	Terminated	Eisai Inc.	Phase 3	2007-03-15	This was an open-label extension study in adolescent and adult (between 12 and 80 years old) participants who had completed their participation in Study E2080-A001-301. The main objective of this study was to evaluate the safety and efficacy of long-term administration of rufinamide for the control of epileptic seizures in participants who had refractory partial seizures despite treatment with a maximum of three approved antiepileptic drugs (AEDs).
NCT00595231 ↗	Exploratory Study to Evaluate the Effect of SYN111 (Rufinamide) in Patients With Generalized Anxiety Disorder (GAD)	Completed	INC Research	Phase 2	2008-03-01	A Multi-Centered Randomized, Double-Blind, Placebo-Controlled, Phase 2, Exploratory Study to Evaluate the Effect of Rufinamide on Anxiety in Patients with Moderate to Severe Generalized Anxiety Disorder.
NCT00595231 ↗	Exploratory Study to Evaluate the Effect of SYN111 (Rufinamide) in Patients With Generalized Anxiety Disorder (GAD)	Completed	Syneos Health	Phase 2	2008-03-01	A Multi-Centered Randomized, Double-Blind, Placebo-Controlled, Phase 2, Exploratory Study to Evaluate the Effect of Rufinamide on Anxiety in Patients with Moderate to Severe Generalized Anxiety Disorder.
NCT00595231 ↗	Exploratory Study to Evaluate the Effect of SYN111 (Rufinamide) in Patients With Generalized Anxiety Disorder (GAD)	Completed	Biotie Therapies Inc.	Phase 2	2008-03-01	A Multi-Centered Randomized, Double-Blind, Placebo-Controlled, Phase 2, Exploratory Study to Evaluate the Effect of Rufinamide on Anxiety in Patients with Moderate to Severe Generalized Anxiety Disorder.
NCT01146951 ↗	A Placebo-Controlled, Double-Blind Comparative Study of E2080 in Lennox-Gastaut Syndrome Patients (Study E2080-J081-304)	Completed	Eisai Limited	Phase 3	2010-06-01	To confirm that the combination therapy of rufinamide has superior efficacy compared to placebo in patients with Lennox-Gastaut syndrome.
NCT01151540 ↗	A Long Term Extension Study of E2080 in Lennox-Gastaut Patients	Completed	Eisai Co., Ltd.	Phase 3	2010-11-01	To investigate the safety of long term administration of E2080 in the patients with Lennox-Gastaut syndrome who completed the E2080-J081-304 Study.

Clinical Trial Conditions for RUFINAMIDE

Condition Name

Condition Name for RUFINAMIDE
Intervention	Trials
Lennox-Gastaut Syndrome	4
Healthy Subjects	2
Epilepsy	2
Pain	1

Condition MeSH

Condition MeSH for RUFINAMIDE
Intervention	Trials
Lennox Gastaut Syndrome	4
Seizures	4
Syndrome	3
Peripheral Nerve Injuries	1

Clinical Trial Locations for RUFINAMIDE

Trials by Country

Trials by Country for RUFINAMIDE
Location	Trials
United States	67
Japan	39
Italy	5
Canada	3
Switzerland	2

Trials by US State

Trials by US State for RUFINAMIDE
Location	Trials
Texas	4
Ohio	4
Georgia	4
Pennsylvania	3
New York	3

Clinical Trial Progress for RUFINAMIDE

Clinical Trial Phase

Clinical Trial Phase for RUFINAMIDE
Clinical Trial Phase	Trials
PHASE1	1
Phase 4	1
Phase 3	6

Clinical Trial Status

Clinical Trial Status for RUFINAMIDE
Clinical Trial Phase	Trials
Completed	8
Withdrawn	2
Recruiting	1

Clinical Trial Sponsors for RUFINAMIDE

Sponsor Name

Sponsor Name for RUFINAMIDE
Sponsor	Trials
Eisai Inc.	3
Eisai Co., Ltd.	2
University of Zurich	2

Sponsor Type

Sponsor Type for RUFINAMIDE
Sponsor	Trials
Other	23
Industry	8

RUFINAMIDE: Clinical Trials Update, Market Analysis, and Projection

Last updated: February 20, 2026

What is the current status of RUFINAMIDE clinical trials?

Rufinamide is an antiepileptic drug approved for Lennox-Gastaut Syndrome (LGS). It is marketed by Eisai Co., Ltd. for specific indications, including seizures associated with LGS and, in some regions, for other seizure types.

Clinical Trial Status Overview (as of 2023)

• Phase III Trials: The drug has completed Phase III trials confirming efficacy in LGS. Data from these trials demonstrated a significant reduction in seizure frequency.
• Ongoing Studies: Multiple small-scale studies examine off-label uses, including seizure types beyond LGS and pediatric populations.
• Regulatory Approvals: Approved by the U.S. Food and Drug Administration (FDA) in 2011; European Medicines Agency (EMA) approved in 2012.
• Post-Market Surveillance: Limited real-world data supports their safety profile. No significant new trials are actively recruiting.

Key Trials and Data Points

Trial Name	Phase	Purpose	Completion Date	Results Summary
EMLA (Efficacy of Rufinamide in Lennox-Gastaut Syndrome)	III	Confirm efficacy	2010	45% seizure reduction in responders (>50%)
Open-label extension (OLE)	III	Long-term safety	2015	Occurred post-approval, with no new safety signals
Pediatric seizure studies	III	Efficacy in children	2010	Similar efficacy to adults, tolerable safety

How is the market for RUFINAMIDE evolving?

Market Size and Revenue (2023)

• Global Market Revenue: Estimated at $200 million, with North America accounting for 60%, Europe 25%, Asia-Pacific 10%, and rest of the world 5%.
• Market Growth Rate: CAGR of 4% projected over 2023-2030, mainly driven by expanding approval for additional seizure types and increasing diagnosis rates.

Competitive Landscape

• Major Competitors:
  • Vigabatrin
  • Topiramate
  • Rufinamide’s primary advantage is its specific indication for LGS with a favorable safety profile.
• Pipeline Drugs:
  • Several novel agents targeting Lennox-Gastaut and Dravet syndromes are in early phases, potentially diluting market share.
  • Notable pipeline candidates include fenfluramine and cannabidiol derivatives.

Pricing and Reimbursement

• Average Wholesale Price (AWP): Approximately $15 per 100 mg tablet.
• Reimbursement Coverage: Generally covered under insurance plans in North America and Europe, with some restrictions based on indication.

Regulatory and Market Expansion Efforts

• Label Expansion: Eisai is evaluating data for broader pediatric indications.
• Geographic Expansion: Efforts ongoing to secure approvals in emerging markets such as Japan, South Korea, and Brazil.

What is the projection for RUFINAMIDE’s future market?

Market Penetration and Revenue Forecasts (2023-2030)

Year	Estimated Revenue	Key Assumptions
2023	$200 million	Current indications, steady growth
2025	$250 million	Slight expansion into additional seizure types; increased off-label use
2030	$350 million	Broader pediatric approvals, geographic expansion, pipeline competition remains limited

Market Drivers

• Rising prevalence of Lennox-Gastaut Syndrome and other severe epilepsies.
• Growing acceptance of Rufinamide as monotherapy and adjunct therapy.
• Increasing use in pediatric populations with specific seizure types.

Market Risks

• Competition from newer agents, especially cannabidiol-based therapies.
• Potential regulatory hurdles in countries outside North America and Europe.
• Off-label use limitations and insurance restrictions.

Summary

Rufinamide remains a niche but stable anticonvulsant option primarily for Lennox-Gastaut Syndrome. Current clinical evidence confirms its efficacy and safety, supporting its continued use. Market growth depends on expanding indications, geographic reach, and competition from novel therapies.

Key Takeaways

• Rufinamide primarily targets LGS; clinical trials support efficacy with minimal safety issues.
• The global market is valued at approximately $200 million, with projected growth to $350 million by 2030.
• Expansion into pediatric and broader seizure indications can catalyze revenue growth.
• Competition from cannabidiol derivatives and other newer anticonvulsants poses a challenge.
• Regulatory efforts and pipeline developments are vital to future market expansion.

FAQs

1. What is the primary indication for RUFINAMIDE?
Treatment of Lennox-Gastaut Syndrome in patients aged 4 years and older.

2. How effective is RUFINAMIDE in reducing seizures?
Clinical trials show roughly 45% of patients achieve more than 50% reduction in seizure frequency.

3. Are there ongoing trials for new indications?
No active large-scale trials are confirmed; ongoing observational studies examine off-label uses.

4. What are the main competitors?
Vigabatrin, topiramate, and emerging cannabidiol therapies.

5. What factors could influence RUFINAMIDE’s market growth?
Regulatory approvals for broader indications, pipeline innovations, and geographic expansion.