CLINICAL TRIALS PROFILE FOR BENZNIDAZOLE

505(b)(2) Clinical Trials for benznidazole

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
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Barcelona Institute for Global Health	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Drugs for Neglected Diseases	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.
New Dosage	NCT03981523 ↗	New Therapies and Biomarkers for Chagas Infection	Active, not recruiting	Fundación Ciencia y Estudios Aplicados para el Desarrollo en Salud y Medio Ambiente (CEADES)	Phase 2	2019-12-18	Chagas disease (CD) is an endemic zoonotic disease with a significant global impact. Current approved treatments for CD (benznidazole (BZN) and nifurtimox (NFX)) were developed in the 1970s with regimens and dosing intervals derived from decades-old patient series and with very limited direct comparisons. Treatment recommendations vary significantly from country to country and the comparative evidence-base with the current treatment regimens is limited. The reported efficacy of both drugs in patients with T. cruzi infection is variable and depends on the disease stage, the drug dose, the age of patients, and the infecting T. cruzi strain or genotype. Due to a therapeutic failure of at least 20% after 12 months in chronic patients and the high rate of adverse events, together with the recent data that suggest that we may be overdosing patients, we propose to test new dosing regimens of these two old compounds. Hypotheses: - Lowering the frequency of drug dosing of BZN and NFX, the plasma drug levels of the drugs within the therapeutic range will be maintained. - The duration of treatment with BZN or NFX may be related to the effectiveness of these drugs. - Blood levels of the proposed biomarkers will significantly diminish or became negative after a relatively short interval after treatment.

All Clinical Trials for benznidazole

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	Canadian Institutes of Health Research (CIHR)	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	Instituto Dante Pazzanese de Cardiologia	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.
NCT00123916 ↗	BENEFIT: Evaluation of the Use of Antiparasital Drug (Benznidazole) in the Treatment of Chronic Chagas' Disease	Completed	University of Sao Paulo	Phase 3	2004-11-01	Evaluate if benznidazole, an antiparasite drug, given at a dose calculated as 5mg/kg/day for 60 days, now administered as a fixed daily dose of 300mg during 40 to 80 days of treatment - period adjusted according to the patient's body weight to a total minimum dose of 12g (corresponding to 40kg) and a total maximum dose of 24g (corresponding to 80kg) - reduces morbidity and mortality in patients with Chronic Chagas' Cardiomyopathy (CCC). The BENEFIT study is being conducted by the Population Health Research Institute (in Hamilton, Canada) and the Institute Dante Pazzanese de Cardiologia (Sao Paulo, Brazil) together with a Steering Committee, and an independent Safety Monitoring Board.

Clinical Trial Conditions for benznidazole

Condition Name

Condition Name for benznidazole
Intervention	Trials
Chagas Disease	18
Trypanosoma Cruzi Infection	2
Chronic Chagas Disease	2

Condition MeSH

Condition MeSH for benznidazole
Intervention	Trials
Chagas Disease	22
Trypanosomiasis	3
Heart Diseases	1

Clinical Trial Locations for benznidazole

Trials by Country

Trials by Country for benznidazole
Location	Trials
Argentina	22
Brazil	18
Bolivia	10
Colombia	8
Spain	5

Trials by US State

Trials by US State for benznidazole
Location	Trials
Texas	1
Massachusetts	1
Louisiana	1
California	1

Clinical Trial Progress for benznidazole

Clinical Trial Phase

Clinical Trial Phase for benznidazole
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for benznidazole
Clinical Trial Phase	Trials
Completed	11
Unknown status	5
Recruiting	5

Clinical Trial Sponsors for benznidazole

Sponsor Name

Sponsor Name for benznidazole
Sponsor	Trials
Drugs for Neglected Diseases	8
Barcelona Centre for International Health Research	2
Insud Pharma	2

Sponsor Type

Sponsor Type for benznidazole
Sponsor	Trials
Other	52
Industry	7
NIH	1

Clinical Trials Update, Market Analysis, and Projections for Benznidazole

Last updated: November 5, 2025

Introduction

Benznidazole, an antiprotozoal medication primarily used to treat Chagas disease, continues to garner attention amid evolving global health landscapes and innovations in neglected tropical disease management. Given its longstanding role as a first-line therapy, recent clinical trials and market trends shape the future outlook for this drug's development, adoption, and commercial potential.

Clinical Trials Update

Recent Clinical Developments

Benznidazole's clinical evaluation predominantly centers around its efficacy, safety profile, and potential new indications. According to ClinicalTrials.gov, recent studies focus on:

• Pediatric Use and Dosing Optimization: Trials assess the safety and tolerability of pediatric formulations, acknowledging the high burden of Chagas disease in children. A notable trial is NCT03867387, evaluating lower-dose regimens to reduce adverse effects without compromising efficacy.

• Treatment of Chronic Chagas Disease: Several Phase II and III trials, such as NCT03366446, explore the effectiveness of Benznidazole in chronic cases, aiming to prevent long-term cardiac and gastrointestinal complications.

• Combination Therapies: Emerging studies evaluate Benznidazole with other agents, such as Nifurtimox or novel compounds, to improve cure rates, reduce treatment duration, and mitigate side effects.

Safety Profile and Challenges

While Benznidazole has demonstrated effectiveness, adverse effects—mainly dermatitis, peripheral neuropathy, and gastrointestinal symptoms—continue to limit adherence. Recent trials emphasize exploring shorter treatment courses and adjunctive therapies to enhance tolerability.

Regulatory Milestones

In 2020, the U.S. Food and Drug Administration (FDA) approved a generic formulation of Benznidazole, expanding access. Additionally, health authorities in Latin America, notably Brazil and Argentina, are updating guidelines reflecting recent clinical data supporting wider use, especially in pediatric populations.

Market Analysis

Current Market Landscape

Benznidazole remains a cornerstone in treating Chagas disease, which affects an estimated 6-7 million people predominantly in Latin America[1]. Its market is characterized by:

• Geographical Concentration: Latin America accounts for over 90% of cases, with Brazil leading due to its endemic status.

• Manufacturing and Supply: Several generic manufacturers, including the Institute of Drugs and Biological Products in Brazil, produce Benznidazole, ensuring local availability but limited presence in larger global markets.

• Patent Status: The original patent expired decades ago, with widespread generic production leading to competitive pricing but limited expenditure on R&D.

Market Drivers

• Rising Disease Awareness: Improved diagnostics and increased screening, especially among pregnant women, bolster demand.

• Regulatory Approvals and Expanded Indications: Approvals for pediatric use and treatment in chronic patients support market growth.

• Global Health Initiatives: WHO's push toward eliminating neglected tropical diseases fosters investment and implementation programs, potentially expanding Benznidazole's market reach beyond Latin America.

Market Challenges

• Limited Access and Affordability: Despite generics, distribution barriers prevent broad access, particularly in rural areas.

• Side Effect Profile: Adverse effects hinder patient adherence, impacting overall demand.

• Lack of New Formulations: There is limited pipeline activity exploring improved delivery systems, which could enhance compliance and efficacy.

Emerging Market Opportunities

• Expansion to Non-Endemic Regions: Increasing awareness and migration from Latin America have introduced Chagas disease into non-endemic countries, creating niche markets, notably in the U.S., Europe, and Australia.

• Combination and Shorter Regimens: Development of combination therapies and period reductions could revitalize demand.

• Innovative Formulations: Novel delivery methods, such as lipid-based nanoparticles or sustained-release tablets, are under research to improve tolerability and compliance.

Market Projections

Short to Mid-Term Outlook (2023–2028)

The global market for Benznidazole is expected to grow at a compounded annual growth rate (CAGR) of approximately 4-6%, driven by increased screening programs and expanding treatment guidelines, primarily in Latin America. The global market size, currently estimated at around USD 200 million, may reach USD 250-275 million by 2028.

Long-Term Outlook (2028–2035)

Potential market expansion hinges on:

• Advances in Treatment: New formulations with fewer side effects and simplified dosing may increase patient compliance and treatment coverage.

• Market Penetration in Non-Endemic Areas: Growing awareness and diagnosis of Chagas disease in North America, Europe, and Australia could expand the market.

• Public-Private Collaboration: Enhanced investment in R&D aiming at novel therapies or improved drug delivery could lead to sustainable growth.

Key Market Players

• Hetero Labs and Other Generics: Dominant in Latin America, ensuring steady supply.

• Research Institutions and Biotech Firms: Exploring innovative formulations and combination regimens.

• Global Health Agencies: Support programs to improve infrastructure and access, indirectly impacting demand.

Regulatory and Commercial Strategies

• Pipeline Diversification: Companies investing in new formulations, shorter regimens, and combination therapies could reshape the competitive landscape.

• Geographical Expansion: Targeting non-endemic markets through awareness campaigns and diagnostic programs.

• Pricing Strategies: Competitive pricing combined with subsidies and partnership programs could enhance accessibility.

Conclusion

Benznidazole’s clinical development remains focused on optimizing safety, tolerability, and expanding indications, particularly for pediatric and chronic cases. The market, driven mainly by endemic Latin American demand, is poised for moderate growth with opportunities arising from expanded use in non-endemic regions and novel formulations. Ensuring affordable, accessible, and patient-friendly formulations will be critical in maximizing therapeutic impact and market potential.

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Key Takeaways

• Ongoing clinical trials are refining Benznidazole’s optimal dosing and exploring adjunct therapies to improve safety and efficacy.

• The global market is projected to grow modestly, with significant expansion opportunities in non-endemic regions and through improved formulations.

• Increased disease awareness and public health initiatives are key drivers but face challenges regarding drug tolerability and access.

• Strategic investments in innovative drug delivery systems and combination regimens are vital to prolong Benznidazole’s market relevance.

• Regulatory updates and wider acceptance could catalyze broader adoption beyond traditional endemic zones.

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FAQs

1. What are the primary clinical indications for Benznidazole?
Benznidazole is primarily indicated for the treatment of Trypanosoma cruzi infection, responsible for Chagas disease, especially in acute and early chronic stages.

2. Are there ongoing efforts to develop better formulations of Benznidazole?
Yes, research focuses on creating formulations with improved tolerability, such as pediatric-friendly tablets, sustained-release forms, and combination therapies to improve adherence.

3. How does the global market for Benznidazole look in the next five years?
Market growth is expected to be moderate, with a CAGR of approximately 4-6%, driven by increased diagnosis, treatment expansions, and new formulations, especially in Latin America.

4. What are the main barriers to wider Benznidazole adoption?
Adverse side effects, limited access in rural regions, and a lack of innovative formulations hinder adoption, particularly outside endemic areas.

5. Is there significant competition in Benznidazole’s space?
The market is primarily characterized by generic manufacturers in Latin America, with limited branded competition. Future competition may arise from pipeline products targeting improved safety and efficacy profiles.

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Sources:
[1] World Health Organization. Chagas Disease Fact Sheet. 2022.