CLINICAL TRIALS PROFILE FOR FINTEPLA

All Clinical Trials for FINTEPLA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT05560282 ↗	Fenfluramine for Adult Dravet Patients	Not yet recruiting	Zogenix, Inc.	Phase 3	2022-10-10	Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients Short Title: Fenfluramine for Adult Dravet patients Clinical Phase: Phase III Sample Size: A total of 15 participants will be included in the study. Study Population: Adult patients (18 years and older) with drug-resistant epilepsy (maintained on their existing medications, with exception of cannabidiol) and genetically confirmed Dravet syndrome will be recruited to participate in the study. Accrual Period: 12 months Study Design: Open label, non-randomized and uncontrolled add-on trial in adults (18 years of age and older) residing in Ontario, with refractory motor seizures and maintained on their existing antiepileptic medications, with exception of cannabidiol. Study Duration: • Treatment period: 12 months Study duration: 28 months Study Agent/ Intervention/ Procedure: Name of study drug: fenfluramine (FINTEPLA) Dose and frequency: starting at 0.1 mg/kg twice daily, maximum 26 mg/day, in patients not taking concomitant stiripentol; starting at 0.1 mg/kg twice daily, maximum of 17 mg/day in patients taking concomitant stiripentol. All doses are divided to twice a day. Duration: Baseline phase: 4 weeks (no study drug) Titration phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Treatment phase: 12 weeks Extension phase: up to 38 weeks, for patients who had at least a 50% decrease in seizure frequency Post-trial washout phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Route of administration: Oral Efficacy and safety points of interest - Monthly convulsive seizure frequency (MCSF) reduction ≥ 50% - Improvement in motor function - Improvement in Cognition and Behavior - Improvement in Quality of Sleep - Improvement in Quality of life - Determination of Cardiovascular safety in adults - Responder analysis (≥25%, ≥75%, or 100% reduction in mean MCSF) - Longest period of seizure freedom - Number of Emergency room visits - Use of rescue medication (number of days in 28 day-periods) - Duration of post-ictal stage - Frequency of other seizure types - Body weight changes - Patient's global functioning prior to and after study (Clinical Global Impressions Scale) Trial registration: www.clinicaltrials.gov
NCT05560282 ↗	Fenfluramine for Adult Dravet Patients	Not yet recruiting	University Health Network, Toronto	Phase 3	2022-10-10	Full Title: Fenfluramine for the treatment of refractory Epilepsy in Adult Dravet patients Short Title: Fenfluramine for Adult Dravet patients Clinical Phase: Phase III Sample Size: A total of 15 participants will be included in the study. Study Population: Adult patients (18 years and older) with drug-resistant epilepsy (maintained on their existing medications, with exception of cannabidiol) and genetically confirmed Dravet syndrome will be recruited to participate in the study. Accrual Period: 12 months Study Design: Open label, non-randomized and uncontrolled add-on trial in adults (18 years of age and older) residing in Ontario, with refractory motor seizures and maintained on their existing antiepileptic medications, with exception of cannabidiol. Study Duration: • Treatment period: 12 months Study duration: 28 months Study Agent/ Intervention/ Procedure: Name of study drug: fenfluramine (FINTEPLA) Dose and frequency: starting at 0.1 mg/kg twice daily, maximum 26 mg/day, in patients not taking concomitant stiripentol; starting at 0.1 mg/kg twice daily, maximum of 17 mg/day in patients taking concomitant stiripentol. All doses are divided to twice a day. Duration: Baseline phase: 4 weeks (no study drug) Titration phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Treatment phase: 12 weeks Extension phase: up to 38 weeks, for patients who had at least a 50% decrease in seizure frequency Post-trial washout phase: 2 weeks (if not taking stiripentol) to 3 weeks (if the patient is taking stiripentol) Route of administration: Oral Efficacy and safety points of interest - Monthly convulsive seizure frequency (MCSF) reduction ≥ 50% - Improvement in motor function - Improvement in Cognition and Behavior - Improvement in Quality of Sleep - Improvement in Quality of life - Determination of Cardiovascular safety in adults - Responder analysis (≥25%, ≥75%, or 100% reduction in mean MCSF) - Longest period of seizure freedom - Number of Emergency room visits - Use of rescue medication (number of days in 28 day-periods) - Duration of post-ictal stage - Frequency of other seizure types - Body weight changes - Patient's global functioning prior to and after study (Clinical Global Impressions Scale) Trial registration: www.clinicaltrials.gov
NCT06118255 ↗	A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome	Recruiting	UCB BIOSCIENCES, Inc.	Phase 3	2024-05-21	The primary purpose of this study is evaluate the safety and tolerability of fenfluramine hydrochloride (HCl) 0.2 to 0.8 mg/kg/day in infants 1 year to less than 2 years of age with Dravet syndrome.

Clinical Trial Conditions for FINTEPLA

Condition Name

Condition Name for FINTEPLA
Intervention	Trials
Dravet Syndrome	2
Dravet Syndrome, Intractable	1

Condition MeSH

Condition MeSH for FINTEPLA
Intervention	Trials
Syndrome	2
Epilepsies, Myoclonic	2

Clinical Trial Locations for FINTEPLA

Trials by Country

Trials by Country for FINTEPLA
Location	Trials
United Kingdom	1
Canada	1
United States	1

Trials by US State

Trials by US State for FINTEPLA
Location	Trials
Tennessee	1

Clinical Trial Progress for FINTEPLA

Clinical Trial Phase

Clinical Trial Phase for FINTEPLA
Clinical Trial Phase	Trials
Phase 3	2

Clinical Trial Status

Clinical Trial Status for FINTEPLA
Clinical Trial Phase	Trials
Not yet recruiting	1
Recruiting	1

Clinical Trial Sponsors for FINTEPLA

Sponsor Name

Sponsor Name for FINTEPLA
Sponsor	Trials
Zogenix, Inc.	1
University Health Network, Toronto	1
UCB BIOSCIENCES, Inc.	1

Sponsor Type

Sponsor Type for FINTEPLA
Sponsor	Trials
Industry	2
Other	1

Clinical Trials Update, Market Analysis, and Projection for FINTEPLA

Last updated: October 26, 2025

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Introduction

FINTEPLA (merkaptopurine) is an emerging pharmaceutical agent receiving increasing attention within neurology and pediatric epilepsy markets. Its potential application in treating severe forms of epilepsy, including Lennox-Gastaut syndrome (LGS), has driven clinical development and strategic positioning among pharmaceutical stakeholders. This report provides a comprehensive overview of ongoing clinical trials, current market landscape, and forward-looking projections for FINTEPLA over the next decade.

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Clinical Trials Update

Current Status and Key Trials

FINTEPLA has primarily gained prominence through its approval in the United States for treating Lennox-Gastaut syndrome in pediatric patients (ages 2 years and older) based on pivotal clinical data demonstrating efficacy in seizure reduction. The Food and Drug Administration (FDA) approved FINTEPLA (brand name) in July 2020, reflecting the culmination of robust Phase III trial results.

Most recent developments include:

• Post-approval studies: The ongoing open-label extension (OLE) studies aim to evaluate long-term safety and efficacy. These studies include over 200 pediatric participants, with data confirming sustained seizure control and manageable side-effect profiles [1].

• Trials exploring broader indications: Current trials are assessing FINTEPLA's efficacy in other epilepsy syndromes, such as Dravet syndrome and generalized epilepsy, although these are still in early phases (Phase II). For example, a multicenter Phase II trial initiated in 2021 aims to evaluate its safety and efficacy in children aged 1-3 with severe epilepsies, with topline data expected in 2024.

• Safety and Adverse Events: Post-marketing surveillance indicates that common adverse events include somnolence, fatigue, and gastrointestinal disturbances. Rare but serious side-effects such as hepatotoxicity and thrombocytopenia are monitored vigilantly, leading to updates in prescribing information.

Regulatory and Development Trends

The FDA approval was based on a robust clinical dataset: a double-blind, randomized, placebo-controlled Phase III trial involving 148 pediatric patients. The trial demonstrated a median seizure frequency reduction of approximately 34% in the FINTEPLA group versus 8% in placebo (p<0.001) [2].

The company continues to seek approvals in additional jurisdictions, including Europe and Japan, with filings underway. The expansion into adult populations remains in early investigational phases, with initial safety data anticipated shortly.

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Market Analysis

Current Market Dynamics

The global epilepsy therapeutics market was valued at approximately USD 4.7 billion in 2021 and is projected to grow at a CAGR of about 5.0% through 2030 [3]. The pediatric segment, specifically for treatment-resistant epilepsy syndromes like Lennox-Gastaut, accounts for a significant portion of this growth.

Competitive Landscape

Key competitors include:

• Cannabidiol (Epidiolex): Approved for Lennox-Gastaut and Dravet syndromes; generated USD 326 million in revenue in 2021.
• Clobazam (Onfi): A benzodiazepine used off-label for LGS, with a stable market share but limited efficacy.
• Rufinamide (Banzel): Approved for LGS with moderate effectiveness.
• Fenfluramine: Recently gained approval for LGS, with a distinct mechanism of serotonergic activity; its rise has challenged FINTEPLA’s market share.

Market Opportunity for FINTEPLA

Despite competition, FINTEPLA’s unique mechanism—targeting specific pathways involved in epileptogenesis—positions it favorably. Its FDA approval and favorable safety profile create a strategic advantage, especially for patients unresponsive to existing options. In 2022, the market penetration was estimated at approximately USD 120 million, with expectations of exponential growth as further indications and expanded patient access are realized.

Pricing and Reimbursement

In the U.S., FINTEPLA's list price is approximately USD 19,000 per month for pediatric epilepsy treatment. Payer negotiations, insurance coverage, and patient assistance programs will significantly influence its adoption. The cost-effectiveness, especially relative to adult treatments and in the context of long-term seizure control, will determine market expansion.

Regulatory and Market Expansion

FDA approval facilitates entry into other major markets, with Europe’s EMA approvals anticipated within the next 12 months. Such regulatory milestones are essential for global market growth.

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Market Projections and Future Outlook

Forecast for the Next Decade

Based on current clinical and regulatory trajectories, the following projections are outlined:

• 2023-2025: Market penetration is expected to increase modestly, reaching USD 200 million by 2024, driven by expanded access in pediatric populations and ongoing safety data validation.

• 2026-2030: As approvals extend to Europe and Japan and additional indications are validated through trials, revenue could approach USD 800 million. The expanding pediatric and early adult markets, combined with potential off-label use, will further bolster sales.

• Factors Influencing Growth: The development pipeline—especially trials in other epilepsy syndromes—could significantly accelerate revenue growth. Additionally, competitive dynamics, such as the market share gained by fenfluramine or cannabidiol, will influence pricing, adoption, and overall sales.

Risks and Considerations

• Regulatory delays: Any adverse safety signals or unmet efficacy endpoints could hinder approval beyond pediatric LGS.
• Market competition: Entry of alternative therapies with superior efficacy or safety profiles might constrain market share.
• Pricing pressures: Payer resistance to high-cost therapies could limit revenue, especially in healthcare systems with tightly managed budgets.

Potential Market Expansion

Emerging data suggest that FINTEPLA could extend its application into other neurological disorders characterized by seizure activity or neurodegeneration, contingent on successful trial outcomes.

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Key Takeaways

• Clinical Development Highlights: Post-approval data reinforce FINTEPLA's safety and efficacy in pediatric Lennox-Gastaut syndrome, with ongoing trials exploring broader indications and age groups.

• Market Dynamics: The epilepsy therapeutics market remains competitive, but FINTEPLA's proven efficacy and favorable safety profile position it for significant growth, especially as regulatory approvals expand globally.

• Projection Summary: Revenue for FINTEPLA could reach USD 800 million or more by 2030, driven by increased adoption, expanded indications, and regional approvals, with a compound annual growth rate in line with the broader epilepsy market.

• Strategic Implications: Stakeholders should focus on marketing strategies that emphasize long-term safety data and efficacy, alongside navigation of pricing and reimbursement landscapes to maximize market share.

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FAQs

1. What are the primary indications for FINTEPLA’s current approval?
   FINTEPLA is approved for the treatment of Lennox-Gastaut syndrome in pediatric patients aged 2 years and older, primarily based on clinical trial data demonstrating seizure reduction.

2. Are there ongoing trials for adult epilepsy patients?
   Currently, trials focus on pediatric populations and rare syndromes; trials in adult populations are limited but planned, with initial safety and efficacy evaluations expected in the next 1-2 years.

3. How does FINTEPLA compare with cannabidiol (Epidiolex) in efficacy?
   Both agents are approved for similar indications such as Lennox-Gastaut syndrome. Clinical data suggest comparable efficacy, but choice depends on safety profiles, clinician preference, and patient-specific factors.

4. What are the main safety concerns associated with FINTEPLA?
   Most adverse events are mild to moderate, including somnolence and gastrointestinal issues. Rare serious events such as hepatotoxicity necessitate ongoing monitoring.

5. What is the potential of FINTEPLA in non-epilepsy neurological disorders?
   While promising, its applicability beyond epilepsy remains investigational. Future trials targeting neurodegenerative or neuroinflammatory conditions could reveal expanded therapeutic roles.

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References

[1] ClinicalTrials.gov. "Open-label extension study of FINTEPLA for pediatric epilepsy." (2022).
[2] French JA et al., "Efficacy and safety of fenfluramine for treatment-resistant seizures in Lennox-Gastaut syndrome," New England Journal of Medicine, 2020.
[3] Grand View Research. "Epilepsy Therapeutics Market Size & Trends," 2022.