CLINICAL TRIALS PROFILE FOR EPIDIOLEX

505(b)(2) Clinical Trials for EPIDIOLEX

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
OTC	NCT04423341 ↗	Effect of Non-psychoactive Cannabidiol as an Adjunct to Botulinum Toxin in Blepharospasm	Completed	Benign Essential Blepharospasm Research Foundation	Phase 2/Phase 3	2020-05-20	The planned study is a prospective analysis of non-psychoactive Cannabidiol (without THC) as an adjunctive therapy for blepharospasm in a masked double cross-over study. This prospective study is a follow-up to a retrospective study completed by the researchers using over-the-counter, self purchased CBD. This study will use FDA approved Cannabidiol medication, Epidiolex, directly from GW pharmaceuticals, rather than self-purchased CBD from the internet. Patients will undergo videorecording with a high resolution videocamera system at days 0, 45, 90, 135, and 180 using a novel blink analysis to gather objective data measurements of changes induced by CBD in Blepharospasm patients. This study will attempt to codify the data and quantify if adjunctive CBD therapy improves those areas compared to botulinum injection alone.
OTC	NCT04423341 ↗	Effect of Non-psychoactive Cannabidiol as an Adjunct to Botulinum Toxin in Blepharospasm	Completed	GW Pharmaceuticals Ltd.	Phase 2/Phase 3	2020-05-20	The planned study is a prospective analysis of non-psychoactive Cannabidiol (without THC) as an adjunctive therapy for blepharospasm in a masked double cross-over study. This prospective study is a follow-up to a retrospective study completed by the researchers using over-the-counter, self purchased CBD. This study will use FDA approved Cannabidiol medication, Epidiolex, directly from GW pharmaceuticals, rather than self-purchased CBD from the internet. Patients will undergo videorecording with a high resolution videocamera system at days 0, 45, 90, 135, and 180 using a novel blink analysis to gather objective data measurements of changes induced by CBD in Blepharospasm patients. This study will attempt to codify the data and quantify if adjunctive CBD therapy improves those areas compared to botulinum injection alone.
OTC	NCT04423341 ↗	Effect of Non-psychoactive Cannabidiol as an Adjunct to Botulinum Toxin in Blepharospasm	Completed	Smith-Kettlewell Eye Research Institute	Phase 2/Phase 3	2020-05-20	The planned study is a prospective analysis of non-psychoactive Cannabidiol (without THC) as an adjunctive therapy for blepharospasm in a masked double cross-over study. This prospective study is a follow-up to a retrospective study completed by the researchers using over-the-counter, self purchased CBD. This study will use FDA approved Cannabidiol medication, Epidiolex, directly from GW pharmaceuticals, rather than self-purchased CBD from the internet. Patients will undergo videorecording with a high resolution videocamera system at days 0, 45, 90, 135, and 180 using a novel blink analysis to gather objective data measurements of changes induced by CBD in Blepharospasm patients. This study will attempt to codify the data and quantify if adjunctive CBD therapy improves those areas compared to botulinum injection alone.
OTC	NCT04423341 ↗	Effect of Non-psychoactive Cannabidiol as an Adjunct to Botulinum Toxin in Blepharospasm	Completed	Silkiss Eye Surgery	Phase 2/Phase 3	2020-05-20	The planned study is a prospective analysis of non-psychoactive Cannabidiol (without THC) as an adjunctive therapy for blepharospasm in a masked double cross-over study. This prospective study is a follow-up to a retrospective study completed by the researchers using over-the-counter, self purchased CBD. This study will use FDA approved Cannabidiol medication, Epidiolex, directly from GW pharmaceuticals, rather than self-purchased CBD from the internet. Patients will undergo videorecording with a high resolution videocamera system at days 0, 45, 90, 135, and 180 using a novel blink analysis to gather objective data measurements of changes induced by CBD in Blepharospasm patients. This study will attempt to codify the data and quantify if adjunctive CBD therapy improves those areas compared to botulinum injection alone.

All Clinical Trials for EPIDIOLEX

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02006628 ↗	A Study of GWP42003 as Adjunctive Therapy in the First Line Treatment of Schizophrenia or Related Psychotic Disorder	Completed	GW Research Ltd	Phase 2	2014-02-25	A study to compare the change in symptom severity in participants with schizophrenia or related psychotic disorder when treated with GWP42003 or placebo in conjunction with existing anti-psychotic therapy over a period of six weeks.
NCT02091206 ↗	A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1)	Completed	GW Research Ltd	Phase 2	2014-10-22	To evaluate the safety and pharmacokinetics (PK) of multiple doses of GWP42003-P compared with placebo in children with Dravet syndrome.
NCT02091375 ↗	Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1)	Completed	GW Research Ltd	Phase 3	2015-03-30	To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.
NCT02224560 ↗	Efficacy and Safety of GWP42003-P for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults	Completed	GW Research Ltd	Phase 3	2015-06-08	The primary objective of this study was to evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo in participants with Lennox-Gastaut syndrome (LGS).
NCT02224573 ↗	GWPCARE5 - An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet or Lennox-Gastaut Syndromes	Completed	GW Research Ltd	Phase 3	2015-06-01	To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet or Lennox-Gastaut syndromes.
NCT02224690 ↗	A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P; CBD) as Adjunctive Treatment for Seizures Associated With Lennox-Gastaut Syndrome in Children and Adults	Completed	GW Research Ltd	Phase 3	2015-04-28	To evaluate the efficacy of GWP42003-P as adjunctive treatment in reducing the number of drop seizures when compared with placebo, in participants with Lennox-Gastaut Syndrome (LGS).
NCT02224703 ↗	GWPCARE2 A Study to Investigate the Efficacy and Safety of Cannabidiol (GWP42003-P) in Children and Young Adults With Dravet Syndrome	Completed	GW Research Ltd	Phase 3	2015-04-13	To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and young adults with Dravet syndrome.

Clinical Trial Conditions for EPIDIOLEX

Condition Name

Condition Name for EPIDIOLEX
Intervention	Trials
Epilepsy	14
Dravet Syndrome	5
Lennox-Gastaut Syndrome	4
CBD	4

Condition MeSH

Condition MeSH for EPIDIOLEX
Intervention	Trials
Syndrome	11
Epilepsy	9
Seizures	8
Disease	6

Clinical Trial Locations for EPIDIOLEX

Trials by Country

Trials by Country for EPIDIOLEX
Location	Trials
United States	179
United Kingdom	9
Spain	8
Australia	7
Poland	4

Trials by US State

Trials by US State for EPIDIOLEX
Location	Trials
New York	16
California	15
Texas	12
Massachusetts	11
Ohio	9

Clinical Trial Progress for EPIDIOLEX

Clinical Trial Phase

Clinical Trial Phase for EPIDIOLEX
Clinical Trial Phase	Trials
PHASE4	1
PHASE3	3
PHASE2	2

Clinical Trial Status

Clinical Trial Status for EPIDIOLEX
Clinical Trial Phase	Trials
Recruiting	27
Completed	18
Not yet recruiting	13

Clinical Trial Sponsors for EPIDIOLEX

Sponsor Name

Sponsor Name for EPIDIOLEX
Sponsor	Trials
GW Research Ltd	17
University of California, San Diego	5
National Institute on Drug Abuse (NIDA)	5

Sponsor Type

Sponsor Type for EPIDIOLEX
Sponsor	Trials
Other	72
Industry	27
NIH	12

Clinical Trials Update, Market Analysis, and Projection for EPIDIOLEX

Last updated: October 26, 2025

Introduction

EPIDIOLEX (cannabidiol), developed by GW Pharmaceuticals (a subsidiary of Jazz Pharmaceuticals), represents a pioneering advancement in the treatment of rare pediatric epilepsy syndromes. Approved by the FDA in 2018, EPIDIOLEX became the first plant-derived cannabinoid medication approved for medical use in the United States. The following analysis synthesizes current clinical trial activity, market dynamics, and future projections, providing stakeholders with vital insights into EPIDIOLEX’s evolving landscape.

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Clinical Trials Status and Developments

Recent Clinical Trials and Studies

Since FDA approval, EPIDIOLEX has undergone extensive post-marketing studies and additional indications exploration. Notably:

• Expanded Indications for Seizure Disorders: The primary FDA approval covers Lennox-Gastaut syndrome (LGS), Dravet syndrome, and tuberous sclerosis complex (TSC). Ongoing trials are investigating its efficacy in other rare seizure disorders, such as CDKL5 deficiency disorder and Doose syndrome. For example, a phase II trial (NCT04974440) is evaluating safety and efficacy in rare childhood epilepsies, aiming to substantiate broader regulatory approval.

• Long-term Safety and Tolerance: A pivotal open-label extension study (GWPCARE6) has confirmed sustained efficacy and safety in pediatric cohorts over five years, bolstering confidence in long-term use. The study’s positive outcomes strengthen EPIDIOLEX’s positioning for further indication expansion.

• Pharmacokinetics and Dosing Optimization: Multiple pharmacokinetic (PK) studies continue to refine dosage regimens, optimizing therapeutic outcomes while minimizing adverse effects, especially in young children. These include phase I trials assessing bioavailability in varied populations.

Ongoing Clinical Pipeline

While primarily focused on epilepsy spectrum disorders, GW Pharmaceuticals (and affiliates) are exploring additional therapeutic avenues:

• Cannabinoid-based therapies for other neurological conditions: Preliminary trials assess EPIDIOLEX in autism spectrum disorder-associated irritability, implying potential expansion into neuropsychiatric domains.

• Combination Therapies: Studies are evaluating synergistic effects with existing anti-epileptic drugs (AEDs), potentially enhancing efficacy and reducing adverse effects.

Regulatory and Clinical Research Outlook: The current clinical pipeline underscores a proactive approach to broadening EPIDIOLEX’s therapeutic footprint. Future approvals hinge on positive outcomes from ongoing trials, with potential milestones anticipated in 2024–2026.

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Market Analysis

Current Market Landscape

EPIDIOLEX commands a unique position as a first-in-class cannabinoid-based therapy approved by regulatory authorities. The market is characterized by:

• Addressable Population: Epiilepsy syndromes covered by EPIDIOLEX affect an estimated 30,000–40,000 children in the U.S. alone,[1] with global numbers significantly higher, especially considering rare pediatric neurological disorders.

• Pricing Strategy: As a specialty medication, EPIDIOLEX’s annual treatment cost ranges from approximately $32,500 to $45,000, reflecting its high-value positioning given its efficacy and safety profile. The high cost is partly justified by the limited patient population and the drug’s innovative status.

• Market Penetration: Since launch, EPIDIOLEX has gained considerable market share within its target segment, although competition from generic formulations of other cannabinoids and emerging therapies remains a threat.

Competitive Environment

While EPIDIOLEX is the first FDA-approved plant-derived cannabidiol, synthetic alternatives are entering the market, especially in jurisdictions with less restrictive regulatory pathways. Notable competitors include:

• Generic cannabinoids: As patents expire, generic versions could exert downward pressure on prices.

• Emerging Therapies: Innovative drugs targeting epileptic syndromes—such as soticlestat (targeting cholesterol 24-hydroxylase)—may challenge EPIDIOLEX’s dominance.

• Off-label Use and Extemporaneous Preparations: Physicians may prescribe compounded cannabinoids, which can impact brand loyalty and sales.

Market Drivers and Barriers

Drivers:

• Increasing recognition of cannabinoids’ therapeutic potential.
• Growing incidence of treatment-resistant epilepsy.
• Favorable safety profile from clinical trials.

Barriers:

• Regulatory uncertainties in some regions.
• High cost impacting affordability and reimbursement.
• Limited awareness beyond specialized centers.

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Market Projection and Future Outlook

Forecasting Epiodiolex’s Growth (2023-2030)

Based on current data and trajectory, analysts project the global cannabinoid-based epilepsy market will experience a compound annual growth rate (CAGR) of approximately 8–10% over the next decade.

• Revenue Projections: EPIDIOLEX’s global sales are expected to reach $900 million–$1.2 billion by 2030, driven by increased clinical adoption, extension into new indications, and geographic expansion, particularly into Europe and Asia-Pacific.

• Geographic Expansion: Regulatory approval processes are underway in several countries, including Japan and Australia, which could significantly boost sales volumes.

• Market Share Trends: As the clinical evidence base expands and dosing guidelines refine, EPIDIOLEX could solidify its market leadership, especially if its expanded indications are approved.

Potential Risks and Opportunities

Risks:

• Regulatory delays or restrictions.
• Price sensitivity and reimbursement challenges.
• Competition from new, more potent, or cost-effective agents.

Opportunities:

• Indication expansion into other neuropsychiatric disorders.
• Development of combination therapies.
• Strategic partnerships and licensing deals to boost international footprint.

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Conclusion

EPIDIOLEX continues to demonstrate robust clinical efficacy and safety in its approved indications, with ongoing trials poised to expand its therapeutic reach. The global epilepsy treatment market, favorably influenced by rising prevalence and recognition of cannabinoid therapeutics, presents a lucrative outlook. Strategic focus on regulatory approval, market access, and indication expansion will be essential for sustained growth. With a projected revenue CAGR of approximately 8–10%, EPIDIOLEX’s future appears promising within the niche of pediatric epilepsy therapeutics.

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Key Takeaways

• Clinical Development: Ongoing studies aim to broaden EPIDIOLEX’s indications, emphasizing its role in various rare epilepsy syndromes and potential neuropsychological applications.

• Market Dynamics: The medication holds a strong position due to its first-mover advantage, unique formulation, and established safety profile, although competition and regulatory variability pose challenges.

• Growth Outlook: International expansion, indication diversification, and favorable driver trends could propel EPIDIOLEX toward $1 billion in global sales by 2030.

• Strategic Considerations: Stakeholders should monitor regulatory developments, pursue geographic expansion, and consider combination therapy research to enhance market share.

• Impact on Industry: EPIDIOLEX’s success underscores the viability of cannabinoid-based medicines, likely catalyzing further innovation and acceptance in mainstream therapeutics.

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FAQs

1. What are the primary approved indications for EPIDIOLEX?
EPIDIOLEX is FDA-approved for Lennox-Gastaut syndrome, Dravet syndrome, and tuberous sclerosis complex in pediatric patients.

2. Are there ongoing trials to expand EPIDIOLEX’s indications?
Yes, several phase II/III trials are investigating its efficacy in other epilepsy syndromes and neuropsychiatric conditions, which could lead to new approvals.

3. How does EPIDIOLEX compare cost-wise to other treatments?
At approximately $32,500–$45,000 annually, it is priced above many traditional AEDs but reflects its novel cannabinoid formulation and orphan drug status.

4. What is the competitive landscape for EPIDIOLEX?
It faces competition from generic cannabinoids, experimental therapies, and off-label compounded products. Patent expiry and regulatory changes may influence market dynamics.

5. What is the long-term outlook for EPIDIOLEX in the global market?
Projected growth, international expansion, and indication widening support a positive outlook, with revenues potentially surpassing $1 billion by 2030.

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References

1. CDC. "Epilepsy in the United States." Centers for Disease Control and Prevention, 2021.