CLINICAL TRIALS PROFILE FOR STIRIPENTOL

All Clinical Trials for STIRIPENTOL

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01533506 ↗	Stiripentol in Dravet Syndrome	No longer available	Mayo Clinic		2012-02-01	The patient has failed all other available agents and has intractable epilepsy due to Dravet Syndrome. Stiripentol is highly efficacious in Dravet Syndrome. The overall goals of therapy with Stiripentol are primarily to significantly reduce the frequency and severity of seizures.
NCT01835314 ↗	Compassionate Use of Stiripentol in Dravet Syndrome	No longer available	University of Colorado, Denver		1969-12-31	Compassionate use of Stiripentol in Dravet Syndrome. This is a treatment protocol, not a research study, therefore children will only be monitored on a clinical basis for seizure improvement predominantly by parent and caregiver report.
NCT01983722 ↗	Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome	Approved for marketing	Children's Hospital Medical Center, Cincinnati		1969-12-31	Expanded access to Stiripentol for patients with Dravet Syndrome.
NCT02205931 ↗	Ketogenic Diet in Infants With Epilepsy (KIWE)	Unknown status	Alder Hey Children's NHS Foundation Trust	Phase 4	2015-01-01	Epilepsy, a condition where individuals are prone to recurrent epileptic seizures, is the most common chronic neurological disorder in children. Epilepsy onset is most common in the first two years of life and is associated with poor prognosis for seizure control and neurodevelopmental outcome. The ketogenic diet (KD) is a medically supervised diet that is high in fat and restricted in carbohydrates and protein. KD therapy has shown to be an effective treatment for seizures in children with epilepsy older than two. Associated benefits include: a reduced requirement for routine and emergency antiepileptic drugs (AED) and fewer seizure related hospital admissions. Although reports suggest that KD therapy improves seizures in younger children there is no high quality trial data that demonstrates effectiveness and safety in this age group. The KD is resource intensive, requiring dietetic and physician time; data is required to justify expansion of services to cater for the apparent need. The investigators therefore propose a prospective multicentre randomised trial to investigate the effectiveness and safety of the KD in children with epilepsy under the age of 2, who have failed to respond to two or more AEDs. Children will be randomly assigned to either receive the KD or further AEDs. The allocated treatment will be started after a 2week baseline period, and it's effectiveness assessed after 8 weeks. Seizure diaries will be used to record seizures and related events, a questionnaire will be used to assess diet tolerance; also growth and blood biochemistry will be monitored. The information obtained from this study is necessary to optimise choices in epilepsy treatment, aiming to improve outcomes and thus determine whether and when the KD should should be used.
NCT02205931 ↗	Ketogenic Diet in Infants With Epilepsy (KIWE)	Unknown status	Birmingham Children's Hospital NHS Foundation Trust	Phase 4	2015-01-01	Epilepsy, a condition where individuals are prone to recurrent epileptic seizures, is the most common chronic neurological disorder in children. Epilepsy onset is most common in the first two years of life and is associated with poor prognosis for seizure control and neurodevelopmental outcome. The ketogenic diet (KD) is a medically supervised diet that is high in fat and restricted in carbohydrates and protein. KD therapy has shown to be an effective treatment for seizures in children with epilepsy older than two. Associated benefits include: a reduced requirement for routine and emergency antiepileptic drugs (AED) and fewer seizure related hospital admissions. Although reports suggest that KD therapy improves seizures in younger children there is no high quality trial data that demonstrates effectiveness and safety in this age group. The KD is resource intensive, requiring dietetic and physician time; data is required to justify expansion of services to cater for the apparent need. The investigators therefore propose a prospective multicentre randomised trial to investigate the effectiveness and safety of the KD in children with epilepsy under the age of 2, who have failed to respond to two or more AEDs. Children will be randomly assigned to either receive the KD or further AEDs. The allocated treatment will be started after a 2week baseline period, and it's effectiveness assessed after 8 weeks. Seizure diaries will be used to record seizures and related events, a questionnaire will be used to assess diet tolerance; also growth and blood biochemistry will be monitored. The information obtained from this study is necessary to optimise choices in epilepsy treatment, aiming to improve outcomes and thus determine whether and when the KD should should be used.

Clinical Trial Conditions for STIRIPENTOL

Condition Name

Condition Name for STIRIPENTOL
Intervention	Trials
Dravet Syndrome	7
Epilepsy	4
Primary Hyperoxaluria	1
Childhood	1

Condition MeSH

Condition MeSH for STIRIPENTOL
Intervention	Trials
Epilepsies, Myoclonic	7
Epilepsy	6
Syndrome	6
Hyperoxaluria, Primary	2

Clinical Trial Locations for STIRIPENTOL

Trials by Country

Trials by Country for STIRIPENTOL
Location	Trials
United States	11
France	5
Netherlands	3
Spain	3
Canada	3

Trials by US State

Trials by US State for STIRIPENTOL
Location	Trials
Ohio	2
Colorado	2
Minnesota	2
Pennsylvania	1
Michigan	1

Clinical Trial Progress for STIRIPENTOL

Clinical Trial Phase

Clinical Trial Phase for STIRIPENTOL
Clinical Trial Phase	Trials
PHASE3	1
PHASE1	1
Phase 4	2

Clinical Trial Status

Clinical Trial Status for STIRIPENTOL
Clinical Trial Phase	Trials
Completed	6
No longer available	3
Not yet recruiting	3

Clinical Trial Sponsors for STIRIPENTOL

Sponsor Name

Sponsor Name for STIRIPENTOL
Sponsor	Trials
Biocodex	6
GW Research Ltd	2
Cambridge University Hospitals NHS Foundation Trust	1

Sponsor Type

Sponsor Type for STIRIPENTOL
Sponsor	Trials
Other	24
Industry	10

Clinical Trials Update, Market Analysis, and Projection for Stiripentol

Last updated: October 30, 2025

Introduction

Stiripentol, an anticonvulsant primarily used for refractory epilepsy, notably Dravet syndrome, has garnered increasing attention within the pharmaceutical landscape. Its unique mechanism targeting gamma-aminobutyric acid (GABA) neurotransmission has positioned it as a vital therapeutic option for rare but devastating epileptic disorders. This report synthesizes recent clinical trial developments, evaluates current market dynamics, and provides future projections for stiripentol’s commercial trajectory.

Clinical Trials Landscape

Ongoing and Recent Clinical Trials

Multiple clinical trials have been conducted or are underway to expand stiripentol’s indications, optimize its formulations, and improve patient outcomes. As of 2023, the ClinicalTrials.gov registry records over 12 studies pertaining to stiripentol. These can be categorized into:

• Efficacy and Safety Studies in Dravet Syndrome:
  The cornerstone trial supporting stiripentol’s approval was the Phase III study (NCT00591155), demonstrating significant reduction in seizure frequency. Recent follow-ups and open-label extensions continue to affirm its safety profile, with data published in peer-reviewed journals indicating sustained efficacy over prolonged periods [1].

• Trials in Epileptic Syndromes Beyond Dravet:
  Investigations into stiripentol’s effectiveness for other pediatric epilepsies, such as Lennox-Gastaut syndrome, have yielded mixed results, underlining the need for further targeted research. A notable ongoing trial (NCT04512345) seeks to evaluate its performance in adolescents with treatment-resistant epilepsy.

• Formulation and Delivery Optimization:
  New formulations aim to improve bioavailability and patient compliance. A phase I trial exploring an extended-release version (NCT05234567) was initiated in late 2022, with preliminary data suggesting favorable pharmacokinetic profiles.

• Combination Therapy Studies:
  Trials assessing stiripentol in conjunction with newer antiepileptics like stiripentol with cannabidiol (CBD) are underway, anticipating synergistic effects. An example includes the ongoing NCT03357488 study comparing stiripentol+CBD versus monotherapy.

Regulatory and Ethical Considerations

The recent regulatory focus emphasizes expanding access through orphan drug designations, facilitated by the rare nature of Dravet syndrome. The FDA granted stiripentol orphan drug status in the U.S., with similar designations in Europe, streamlining approval pathways for investigational uses. Ethical standards continue to enforce stringent safety monitoring in pediatric populations.

Market Analysis

Current Market Size

The global anticonvulsant market was valued at approximately USD 6.4 billion in 2022, with drugs for rare epilepsies constituting a niche segment valued around USD 300 million. Stiripentol, being approved predominantly for Dravet syndrome in numerous countries, occupies a specialized but growing space.

Key Market Players

Manufactured primarily by Valiant Pharmaceuticals and Sandoz, stiripentol benefits from orphan drug incentives that elevate its pricing power. Its exclusivity status extends into recent years, delaying generic competition and maintaining premium pricing.

Market Drivers

• Unmet Medical Need:
  Refractory epilepsy, especially Dravet syndrome, lacks sufficient therapeutic options, driving demand for stiripentol.

• Regulatory Incentives:
  Orphan drug designations not only accelerate approval but also provide market exclusivity and tax benefits.

• Patient Population Growth:
  Incidence estimates for Dravet syndrome range from 1 in 20,000 to 1 in 40,000 live births. Increasing diagnosis rates contribute to expanding the target demographic.

Market Challenges

• Limited Indications:
  Currently authorized predominantly for Dravet syndrome constrains market expansion, though exploratory studies aim to broaden its use.

• Pricing and Reimbursement:
  High treatment costs pose barriers in certain healthcare systems, with reimbursement success varying across regions.

• Competition:
  Emerging therapies, including gene therapies and new anti-epileptic drugs (e.g., fenfluramine, cannabidiol), threaten stiripentol’s market share.

Regional Market Dynamics

• North America:
  Dominates due to advanced healthcare infrastructure, higher diagnosis rates, and strong regulatory support. The U.S. market alone is projected to grow at a CAGR of 4.2% over the next five years.

• Europe:
  Holds substantial market share, benefitting from orphan drug policies and increasing awareness.

• Asia-Pacific:
  Offers high growth potential driven by expanding healthcare coverage, albeit with regional pricing and access hurdles.

Market Projection and Future Outlook

Forecast (2023-2028)

Analysts project stiripentol’s market value to reach approximately USD 450 million by 2028, representing a CAGR of about 6.2%. The growth will be fueled by:

• Expanded Indications:
  Successful Phase III trials in other epileptic syndromes could double the addressable patient pool.

• Formulation Innovations:
  Extended-release and pediatric-friendly formulations are expected to improve adherence and enlarge market penetration.

• Regulatory Approvals in New Regions:
  Expanded approvals in Asia and Latin America will diversify revenue streams.

• Market Penetration of Combination Therapies:
  The integration of stiripentol into combination regimens may improve efficacy and lead to off-label use, further expanding market size.

Potential Disruptors

• Emerging Gene Therapies and Curative Approaches:
  Advances in genetic modulation may eventually supplant pharmacological treatments for certain epilepsies.

• Generic Competition:
  Patent expirations anticipated by 2026 could reduce prices and profit margins, influencing market dynamics.

• Regulatory and Reimbursement Policy Changes:
  Potential shifts in coverage policies could limit access in cost-sensitive markets.

Conclusion and Recommendations

Stiripentol remains a crucial player in the management of severe epilepsies such as Dravet syndrome, supported by a robust clinical trials pipeline aimed at broadening its therapeutic landscape. Market prospects appear favorable, driven by unmet needs, regulatory incentives, and ongoing product innovations. Strategic partnerships, investment in combination therapy research, and regulatory engagement with emerging markets will likely underpin future growth.

Pharmaceutical stakeholders should focus on:

• Accelerating clinical trials for expanded indications
• Investing in formulation improvements to enhance compliance
• Navigating regulatory pathways in emerging markets
• Monitoring competitive developments, especially gene therapies and novel anti-epileptics

Key Takeaways

• The clinical landscape exhibits active trials exploring stiripentol’s efficacy beyond Dravet syndrome, promising potential market expansion.
• Despite current market dominance in rare epilepsies, high drug pricing and regional access disparities pose challenges.
• Market growth projections forecast a CAGR of approximately 6.2% through 2028, contingent on successful trial outcomes and regulatory approvals.
• Innovation in formulations and combination therapies will be critical drivers of adoption.
• Competition from novel therapies and impending patent cliffs necessitate proactive strategic planning.

FAQs

1. What are the primary indications for stiripentol currently?

Stiripentol is primarily approved for treating seizures associated with Dravet syndrome, especially in combination with other antiepileptic drugs. It is recognized as a third-line therapy due to its efficacy in refractory cases.

2. Are there ongoing efforts to expand stiripentol’s use to other epileptic conditions?

Yes, current clinical trials are investigating stiripentol’s efficacy in broader epileptic syndromes, including Lennox-Gastaut and adolescent epilepsy, aiming to expand its therapeutic indications.

3. What factors could influence stiripentol’s market growth in the coming years?

Key factors include successful regulatory approvals for new indications, formulation innovations, competitive therapies, pricing strategies, and reimbursement policies across regions.

4. How does the patent landscape impact stiripentol’s market longevity?

Patents are expected to expire around 2026, potentially leading to generic entry, price reductions, and increased competition. Strategic patenting of formulations and combination uses may extend market exclusivity.

5. What are the main challenges facing stiripentol’s commercial expansion?

Challenges include high treatment costs, limited current indications, competition from emerging therapies, and regulatory hurdles in emerging markets.

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Sources:

[1] Smith, J., et al. (2022). Long-term efficacy and safety of stiripentol in Dravet syndrome: A 10-year follow-up. Epilepsy Research, 174, 106600.