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
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).
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.
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.
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.

Clinical Trial Conditions for Epidiolex

Condition Name

Condition Name for Epidiolex
Intervention	Trials
Epilepsy	14
Dravet Syndrome	5
Lennox-Gastaut Syndrome	4
Healthy	3

Condition MeSH

Condition MeSH for Epidiolex
Intervention	Trials
Syndrome	11
Epilepsy	8
Seizures	7
Disease	6

Clinical Trial Locations for Epidiolex

Trials by Country

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−

Trials by Country for Epidiolex
Location	Trials
United States	171
United Kingdom	9
Spain	8
Australia	7
Poland	4

Trials by US State

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−

Trials by US State for Epidiolex
Location	Trials
California	14
New York	14
Texas	11
Massachusetts	11
Ohio	9

Clinical Trial Progress for Epidiolex

Clinical Trial Phase

Clinical Trial Phase for Epidiolex
Clinical Trial Phase	Trials
Phase 4	10
Phase 3	10
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for Epidiolex
Clinical Trial Phase	Trials
Recruiting	22
Completed	17
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
Hurd,Yasmin, Ph.D.	3

Sponsor Type

Sponsor Type for Epidiolex
Sponsor	Trials
Other	59
Industry	25
NIH	6

Epidiolex: Clinical Trials, Market Analysis, and Projections

Introduction to Epidiolex

Epidiolex, also known as cannabidiol oral solution (GWP42003-P), is a prescription, plant-derived cannabis-based medicine approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). It is indicated for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, and tuberous sclerosis complex (TSC) in patients one year of age and older[1][3][4].

Clinical Trials Update

Phase 3 Trials in Japan

A recent Phase 3 open-label, single-arm clinical trial in Japan evaluated the safety and efficacy of Epidiolex for the treatment of Dravet syndrome (DS) and tuberous sclerosis complex (TSC) in pediatric patients. Although the trial did not meet the primary efficacy endpoint of a pre-specified percentage change in indication-associated seizures, numeric improvements were observed in the primary and several secondary endpoints. No new safety signals were identified, and the trial is ongoing to collect additional data[1].

Previous Phase 3 Trials

Several Phase 3 trials have been conducted to assess the efficacy and safety of Epidiolex. For example, the GWPCARE1 trial involved 120 patients with Dravet syndrome and showed that a 20 mg per kg body weight dose of Epidiolex reduced the average number of monthly convulsive seizures by half compared to the pre-treatment period. Additionally, the GWPCARE2 trial compared high and low doses of Epidiolex to a placebo and found significant reductions in convulsive seizures in both dose groups[4].

Long-Term Safety and Efficacy

The GWPCARE5 open-label Phase 3 extension trial monitored side effects, changes in seizure symptoms, and overall improvements in patients’ quality of life over years of Epidiolex use. The three-year interim analysis indicated that long-term use of Epidiolex continued to reduce the frequency of seizures without new safety concerns[4].

Market Analysis

Global Epilepsy Drugs Market

The global epilepsy drugs market is projected to cross $9 billion by 2025, driven by increasing epilepsy prevalence, growing patient awareness programs, government funding, and reimbursement options in European and American countries. The United States dominates this market due to its robust healthcare support system, growing awareness, and increasing per capita healthcare expenditure[2].

Role of Epidiolex

Epidiolex is one of the key players in the epilepsy drugs market, particularly for the treatment of rare epilepsies such as Dravet syndrome and LGS. The drug has shown significant promise and has been approved for use in the U.S. and the European Union. Its market presence is expected to grow as more data from clinical trials becomes available and as regulatory approvals expand to other regions[2][3].

Market Competition

Epidiolex competes with other anti-epilepsy drugs like Vimpat (Lacosamide) and Keppra (Levetiracetam), which are currently significant players in the market. However, Epidiolex has a unique position due to its cannabinoid-based mechanism of action and its approval for specific rare epilepsies[2].

Market Projections

Growth in Cannabinoid Pharmaceuticals

The global cannabinoid pharmaceuticals market, which includes Epidiolex, is projected to grow from €900 million in 2021 to €1.8 billion by 2025. Epidiolex, in particular, has already achieved significant sales, reaching over €430 million in 2020 and serving an estimated patient population of more than 20,000[5].

Expansion and Regulatory Approvals

The market for Epidiolex is expected to expand as the drug gains regulatory approvals in new regions, such as Japan, where ongoing trials are collecting data to support potential approvals. The acquisition of GW Pharmaceuticals by Jazz Pharmaceuticals for over €6 billion highlights the potential growth and value of Epidiolex in the pharmaceutical market[1][5].

Challenges and Opportunities

Clinical Trial Setbacks

Despite its successes, the development of Epidiolex has faced setbacks in clinical trials. However, these challenges have not deterred the advancement of the drug, and the company has continued to invest in extensive research to improve treatment outcomes for patients with rare epilepsies[5].

Regulatory Milestones

Epidiolex has achieved several regulatory milestones, including approvals in the U.S. and the European Union. Ongoing engagement with regulatory authorities in other regions, such as Japan, is crucial for expanding its market reach[1][3].

Key Takeaways

• Clinical Trials: Epidiolex has shown efficacy and safety in various Phase 3 trials, although some trials have not met all primary endpoints.
• Market Presence: The drug is a significant player in the global epilepsy drugs market, particularly for rare epilepsies.
• Market Growth: The cannabinoid pharmaceuticals market, including Epidiolex, is projected to grow substantially by 2025.
• Regulatory Approvals: Ongoing efforts to secure approvals in new regions will be crucial for market expansion.
• Challenges and Opportunities: Despite clinical trial setbacks, Epidiolex continues to offer significant opportunities for treating rare epilepsies.

FAQs

What is Epidiolex used for?

Epidiolex is used for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, and tuberous sclerosis complex (TSC) in patients one year of age and older[1][3][4].

What are the common side effects of Epidiolex?

Common side effects include fatigue, fever, vomiting, diarrhea, sleepiness, and abnormal liver enzyme test results. Most of these side effects are mild to moderate in severity[4].

How does Epidiolex compare to other epilepsy drugs?

Epidiolex competes with drugs like Vimpat (Lacosamide) and Keppra (Levetiracetam) but has a unique position due to its cannabinoid-based mechanism of action and approval for specific rare epilepsies[2].

What is the current market size of Epidiolex?

Epidiolex achieved sales of over €430 million in 2020 and serves an estimated patient population of more than 20,000[5].

What are the projections for the global cannabinoid pharmaceuticals market?

The market is projected to grow from €900 million in 2021 to €1.8 billion by 2025, with Epidiolex being a key contributor to this growth[5].

Sources

1. Jazz Pharmaceuticals Provides Update on Cannabidiol Oral Solution Phase 3 Trial in Japan. PR Newswire, 22 Aug 2024.
2. The 2019 Global Epilepsy Drugs Market - $9+ Billion Forecast to 2025. Business Wire, 4 Feb 2019.
3. Global Epidiolex (Cannabidiol) Drug Insight and Market Forecasts to 2030. Business Wire, 11 Sep 2020.
4. Epidiolex (Formerly, GWP42003-P) for Dravet Syndrome. Dravet Syndrome News, 16 Oct 2024.
5. Global Cannabinoid Pharmaceuticals Market to Reach £1.53bn by 2025. Business of Cannabis.