CLINICAL TRIALS PROFILE FOR FINTEPLA

All Clinical Trials for FINTEPLA

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
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

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

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−

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

Trials by US State

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−

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

FINTEPLA (Fenfluramine): Clinical Trials, Market Analysis, and Projections

Introduction to FINTEPLA

FINTEPLA, also known as fenfluramine, is a medication approved for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients aged two years and older. Here, we will delve into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials Overview

Phase III Clinical Trials for Dravet Syndrome

The FDA approval of FINTEPLA for DS was based on two Phase III clinical trials, Study 1 and Study 2. These trials were randomized, double-blind, and placebo-controlled, comparing various doses of FINTEPLA with placebo. The primary efficacy endpoint was the change in the frequency of convulsive seizures every 28 days during the treatment period.

• In Study 1, doses of 0.7 mg/kg/day and 0.2 mg/kg/day of FINTEPLA were compared with placebo in patients not receiving stiripentol.
• In Study 2, a 0.4 mg/kg/day dose of FINTEPLA was compared with placebo in patients receiving stiripentol and either clobazam, valproate, or both.

Both studies showed a statistically significant reduction in convulsive seizure frequency for all FINTEPLA dosage groups compared to placebo[1].

Phase III Clinical Trials for Lennox-Gastaut Syndrome

For LGS, the FDA approval was based on a global, randomized, placebo-controlled Phase III clinical trial enrolling 263 patients. This trial demonstrated that a 0.7 mg/kg/day dose of fenfluramine significantly reduced drop seizure frequency compared to placebo. Approximately 25% of patients taking this dose experienced a decrease in drop seizure frequency by at least 50% every 28 days, with 18% seeing a reduction of more than 50% but less than 75%, and 6% achieving a reduction exceeding 75%[1].

Efficacy in Generalized Tonic-Clonic Seizures

Recent analyses published in Epilepsia have further highlighted the efficacy of FINTEPLA in reducing generalized tonic-clonic seizures (GTCS) and tonic-clonic seizures (TCS) in patients with developmental and epileptic encephalopathies (DEEs). These studies showed that 72% of patients achieved a ≥50% reduction in GTCS or TCS from baseline, with 54% and 29% achieving ≥75% and 100% reduction, respectively[2][5].

Market Analysis

Current Market Position

FINTEPLA is currently approved and marketed for DS and LGS in several regions, including the US, Europe, and Japan. However, it faces significant competition from other drugs, particularly Epidiolex (cannabidiol), which has a first-mover advantage and broader indications.

• In 2021, Epidiolex generated over $650 million in sales, while FINTEPLA generated $74.7 million. This disparity is largely due to Epidiolex's earlier approval and broader market reach, including approval for tuberous sclerosis complex (TSC)-associated seizures[3].

Market Potential

Despite the current sales gap, FINTEPLA has significant market potential, especially with its recent approval for LGS. The patient pool for LGS is approximately 45-50,000 in the US, more than double that of DS. This expanded indication could significantly boost FINTEPLA's market presence[3].

Regional Market Challenges

• Europe: FINTEPLA is commercially available in Germany and France and is under NICE technology appraisal in the UK. The European market has been challenging for both FINTEPLA and Epidiolex, with slow rollout and reimbursement issues[3].
• Japan: FINTEPLA has been approved for DS and an additional application for LGS is under review. The collaboration with Nippon Shinyaku is crucial for its market penetration in Japan[1].

Future Projections

Expanding Indications

UCB is advancing FINTEPLA for seizures due to CDKL5 deficiency, a condition affecting roughly 6,000 patients in the US. Although Marinus Pharma’s Ztalmy has gained an FDA nod for the same indication, FINTEPLA still has potential in this niche market[3].

Competitive Landscape

The competition between FINTEPLA and Epidiolex is expected to intensify. However, FINTEPLA's recent approvals and expanding indications position it as a strong contender. The market dynamics may shift as more data on FINTEPLA's efficacy and safety become available, potentially increasing its market share[3].

Sales Projections

Given the expanded indications and growing awareness, FINTEPLA's sales are expected to increase. The addressable patient pool for LGS and potential approvals for other rare epilepsies could drive significant growth. However, the exact sales projections will depend on various factors, including market penetration, reimbursement policies, and competitive strategies[3].

Key Takeaways

• Clinical Efficacy: FINTEPLA has demonstrated significant reductions in seizure frequency for both DS and LGS, as well as in generalized tonic-clonic seizures.
• Market Position: Despite current sales disparities with Epidiolex, FINTEPLA has strong market potential, especially with its expanded indications.
• Future Growth: Expanding indications, particularly for CDKL5 deficiency, and growing market penetration are expected to drive sales growth.
• Competitive Landscape: The competition with Epidiolex will continue, but FINTEPLA's unique profile and growing evidence base position it as a viable alternative.

FAQs

What is FINTEPLA used for?

FINTEPLA (fenfluramine) is used for the treatment of seizures associated with Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS) in patients aged two years and older.

What were the key findings of the Phase III clinical trials for FINTEPLA?

The Phase III trials showed a statistically significant reduction in convulsive seizure frequency for all FINTEPLA dosage groups compared to placebo, with notable reductions in drop seizure frequency for LGS patients.

How does FINTEPLA compare to Epidiolex in the market?

FINTEPLA currently trails Epidiolex in sales due to Epidiolex's earlier approval and broader indications. However, FINTEPLA's recent approvals and expanding indications are expected to increase its market presence.

What are the common adverse reactions associated with FINTEPLA?

Common adverse reactions include reduced appetite, diarrhea, weariness, somnolence, and vomiting.

Is FINTEPLA approved for other indications besides DS and LGS?

FINTEPLA is being advanced for seizures due to CDKL5 deficiency, although it has not yet been approved for this indication.

Sources

1. Clinical Trials Arena: "FINTEPLA (Fenfluramine) for the Treatment of Seizures Associated with Dravet Syndrome and Lennox-Gastaut Syndrome"[1].
2. PR Newswire: "FINTEPLA® (fenfluramine) Results Examining its Impact in Managing Generalized Tonic-Clonic Seizures in Developmental and Epileptic Encephalopathies Published in Epilepsia"[2].
3. DelveInsight: "Epidiolex vs Fintepla: Fight in Rare Epilepsies"[3].
4. Nasdaq: "FINTEPLA® (fenfluramine) Results Examining its Impact in Managing Generalized Tonic-Clonic Seizures in Developmental and Epileptic Encephalopathies"[4].
5. UCB USA: "FINTEPLA® (fenfluramine) Results Examining its Impact in Managing Generalized Tonic-Clonic Seizures in Developmental and Epileptic Encephalopathies Published in Epilepsia"[5].