CLINICAL TRIALS PROFILE FOR FOMEPIZOLE

All Clinical Trials for fomepizole

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT02415712 ↗	Specified Drug-use Survey of Fomepizole Intravenous Infusion (All-case Surveillance)	Recruiting	Takeda		2015-01-27	The objective of this survey is to evaluate the safety and efficacy of fomepizole intravenous infusion in Japanese patients with ethylene glycol and methanol poisonings in daily medical practice.
NCT00661141 ↗	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism	Completed	Ted Daley, President	Phase 2	2008-04-01	This trial will evaluate if fomepizole (4-methylpyrazole) can treat symptoms associated with alcohol intolerance due to aldehyde dehydrogenase 2 (ALDH2) deficiency, an inherited metabolic disorder. These symptoms include flushing, nausea, headache, shortness of breath and dizziness, resulting from exposure to acetaldehyde, the primary metabolite of ethanol. Long-term, serious health risks have been associated with repeated exposure to acetaldehyde, a carcinogen, among ALDH2-deficient individuals.
NCT00661141 ↗	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism	Completed	Horizon Pharma USA, Inc.	Phase 2	2008-04-01	This trial will evaluate if fomepizole (4-methylpyrazole) can treat symptoms associated with alcohol intolerance due to aldehyde dehydrogenase 2 (ALDH2) deficiency, an inherited metabolic disorder. These symptoms include flushing, nausea, headache, shortness of breath and dizziness, resulting from exposure to acetaldehyde, the primary metabolite of ethanol. Long-term, serious health risks have been associated with repeated exposure to acetaldehyde, a carcinogen, among ALDH2-deficient individuals.
NCT00661141 ↗	Phase IIa Study of Fomepizole for Acetaldehyde Toxicity After Ethanol Exposure in Subjects With Altered Ethanol Metabolism	Completed	Raptor Pharmaceuticals Inc.	Phase 2	2008-04-01	This trial will evaluate if fomepizole (4-methylpyrazole) can treat symptoms associated with alcohol intolerance due to aldehyde dehydrogenase 2 (ALDH2) deficiency, an inherited metabolic disorder. These symptoms include flushing, nausea, headache, shortness of breath and dizziness, resulting from exposure to acetaldehyde, the primary metabolite of ethanol. Long-term, serious health risks have been associated with repeated exposure to acetaldehyde, a carcinogen, among ALDH2-deficient individuals.
NCT00346853 ↗	Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy	Completed	University of Utah	Phase 1	2005-11-01	The purpose of this study is to investigate whether taking 4-methylpyrazole (4-MP, fomepizole, Antizol™) inhibits dark adaptation of the eye. In other words, we are testing if 4-MP slows the processing of vitamin A derivatives in the eye. By slowing down these processes, individuals with Stargardt disease may have better chances of saving their remaining vision. 4-MP has been shown to slow dark adaptation in animals, and is FDA approved for human use to treat individuals with methanol or ethylene glycol (antifreeze) poisoning by shutting down the body's ability to process alcohols. This medication has an excellent safety profile and has been reported to have no short-term or long-term side effects, as long as patients refrain from any alcohol while the medication is in the body. A single dose of 4-MP remains in the body for about 12 hours, and therefore, it may inhibit dark adaptation of your eyes for up to 12 hours. Studying the effects of 4-MP may lead to effective medical treatment to save Stargardt patients' vision, and may also have similar effects in other macular degenerative diseases.

Clinical Trial Conditions for fomepizole

Condition Name

Condition Name for fomepizole
Intervention	Trials
Macular Dystrophy, Corneal	1
Acetaminophen Poisoning	1
Aldehyde Dehydrogenase-2 (ALDH2) Deficiency	1
ALDH2 Deficiency	1

Condition MeSH

Condition MeSH for fomepizole
Intervention	Trials
Poisoning	2
Macular Degeneration	1
Corneal Dystrophies, Hereditary	1
Liver Failure	1

Clinical Trial Locations for fomepizole

Trials by Country

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−

Trials by Country for fomepizole
Location	Trials
United States	4
Japan	1

Trials by US State

+

−

Trials by US State for fomepizole
Location	Trials
Arizona	1
Hawaii	1
California	1
Utah	1

Clinical Trial Progress for fomepizole

Clinical Trial Phase

Clinical Trial Phase for fomepizole
Clinical Trial Phase	Trials
Phase 2	2
Phase 1	1
Early Phase 1	1

Clinical Trial Status

Clinical Trial Status for fomepizole
Clinical Trial Phase	Trials
Completed	3
Not yet recruiting	1
Recruiting	1

Clinical Trial Sponsors for fomepizole

Sponsor Name

Sponsor Name for fomepizole
Sponsor	Trials
University of Utah	1
Ted Daley, President	1
Horizon Pharma USA, Inc.	1

Sponsor Type

Sponsor Type for fomepizole
Sponsor	Trials
Other	4
Industry	4

Fomepizole: Clinical Trials, Market Analysis, and Projections

Introduction

Fomepizole, also known as 4-methylpyrazole, is a critical antidote used in the treatment of ethylene glycol and methanol poisoning. This article delves into the recent clinical trials, market analysis, and future projections for this essential medication.

Clinical Trials and Safety Profile

Recent Clinical Trials in Japan

A significant clinical trial conducted in Japan from January 2015 to June 2022 evaluated the safety and effectiveness of fomepizole in patients with ethylene glycol or methanol poisoning. This retrospective post-marketing surveillance study involved 147 patients across 91 institutions. The results indicated that fomepizole is well tolerated, with no serious adverse drug reactions (ADRs) reported. The most common ADR was vomiting, affecting 2.3% of the patients. The study also showed that fomepizole improved clinical outcomes, with a significant increase in arterial blood pH within 4 hours of treatment, which remained stable for 24 hours post-treatment[1][4].

Longitudinal Cohort Study

A 16-year post-marketing surveillance study further reinforced the safety profile of fomepizole. This longitudinal cohort study supported the safety of fomepizole in treating presumed ethylene glycol (EG) and methanol poisoning, highlighting its efficacy and low incidence of adverse effects[5].

Dosage and Administration

Fomepizole is administered intravenously, with a recommended dosing regimen that includes a loading dose of 15 mg/kg followed by doses of 10 mg/kg every 12 hours for four doses, and then 15 mg/kg every 12 hours thereafter until the toxic substance levels are undetectable or reduced below a certain threshold. The doses should be administered as a slow intravenous infusion over 30 minutes[3].

Market Analysis

Global Market Size and Growth

The global fomepizole market was valued at approximately USD 37 million in 2023 and is projected to reach about USD 61 million by 2033, growing at a Compound Annual Growth Rate (CAGR) of 5.2% during the forecast period from 2024 to 2033. This steady growth is driven by the increasing incidence of methanol and ethylene glycol poisoning and the continued use of fomepizole as a critical antidote in emergency and critical care settings[2].

Market Dynamics

The fomepizole market is characterized by a limited number of key players, as it is a specialized medication with limited competition from generic manufacturers. Ongoing research and development efforts to explore new therapeutic applications and improve formulations are driving market growth. Regulatory approvals and healthcare policies recognizing the role of antidotes in acute poisoning management have also shaped the market dynamics[2].

Regional Market Overview

North America, particularly the United States, is a leading market due to its well-established healthcare infrastructure and the availability of reimbursement for specialized antidotes. Europe is another prominent region, with countries like Germany, the UK, and France playing significant roles. The Asia-Pacific region is expected to experience the fastest growth, driven by increasing awareness and the expansion of healthcare systems in countries like China, India, and Japan. Latin America and the Middle East and Africa also present opportunities for market growth, although the availability and accessibility of fomepizole may be relatively lower in these regions[2].

Market Opportunities and Challenges

Opportunities for Growth

The increasing incidence of methanol and ethylene glycol poisoning, particularly in certain regions or due to specific circumstances, creates a significant demand for fomepizole. The recognition of its role in managing other types of alcohol intoxication or poisoning presents opportunities for market players to explore new therapeutic applications. Emerging markets in Asia-Pacific, Latin America, and the Middle East and Africa offer untapped potential as awareness about fomepizole grows in these regions. Technological advancements, such as novel drug delivery systems, can also enhance the convenience, safety, and efficacy of fomepizole-based treatments[2].

Challenges

Despite the growth prospects, the market faces challenges such as the limited patient population, the high cost of this specialized antidote, and the evolving regulatory landscape. These factors may pose restraints to the market's expansion[2].

Future Projections

Continued Growth

The fomepizole market is poised for continued growth, driven by the persistent need for effective antidotes in emergency and critical care settings. Factors such as the development of improved formulations with enhanced safety profiles, the exploration of new therapeutic applications, and the integration of technological advancements in drug delivery systems will contribute to the market's expansion. The increasing emphasis on personalized and patient-centric approaches to medical treatment, along with the growing adoption of digital health solutions, may also shape the future of the fomepizole market[2].

Regulatory and Research Implications

Regulatory approvals and ongoing research will be crucial in expanding the therapeutic applications of fomepizole. For instance, fomepizole is already recognized by the WHO as an essential medicine and is approved in several countries, including the USA, Canada, Japan, and Ireland. Continued research into its safety and efficacy will further solidify its position in the market[3].

Key Takeaways

• Clinical Safety: Fomepizole has been shown to be well tolerated and effective in treating ethylene glycol and methanol poisoning in clinical trials.
• Market Growth: The global fomepizole market is projected to grow at a CAGR of 5.2% from 2024 to 2033, driven by increasing incidence of poisoning and advancements in healthcare.
• Regional Dynamics: North America and Europe are leading markets, while the Asia-Pacific region is expected to experience the fastest growth.
• Opportunities and Challenges: Emerging markets and technological advancements offer growth opportunities, but the market faces challenges such as high costs and regulatory changes.
• Future Projections: Continued growth is expected, driven by the need for effective antidotes and advancements in healthcare technologies.

FAQs

What is fomepizole used for?

Fomepizole is used as an antidote for the treatment of ethylene glycol and methanol poisoning.

What is the recommended dosing regimen for fomepizole?

The recommended dosing regimen includes a loading dose of 15 mg/kg followed by doses of 10 mg/kg every 12 hours for four doses, and then 15 mg/kg every 12 hours thereafter until the toxic substance levels are undetectable or reduced below a certain threshold.

What are the common adverse reactions associated with fomepizole?

The most common adverse reaction reported is vomiting, affecting approximately 2.3% of patients.

What is the projected growth rate of the fomepizole market?

The fomepizole market is projected to grow at a CAGR of 5.2% from 2024 to 2033.

Which regions are expected to drive the growth of the fomepizole market?

The Asia-Pacific region is expected to experience the fastest growth, driven by increasing awareness and the expansion of healthcare systems in countries like China, India, and Japan.

Sources

1. Real-world safety and effectiveness of intravenous fomepizole in patients with ethylene glycol or methanol poisoning in Japan. PubMed.
2. Fomepizole (4 Methylpyrazole) Market Size, Share, Growth, Trends, Statistics Analysis Report. Data Horizon Research.
3. Australian Public Assessment Report for Fomepizole. Therapeutic Goods Administration.
4. Fomepizole-5001 | Clinical Trial Summary. Takeda Clinical Trials.
5. Analysis of fomepizole safety based on a 16-year post-marketing surveillance. Taylor & Francis Online.