CLINICAL TRIALS PROFILE FOR VORICONAZOLE

505(b)(2) Clinical Trials for VORICONAZOLE

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	NCT02372357 ↗	A New Dosing Regimen for Posaconazole Prophylaxis in Children Based on Body Surface Area	Completed	Institutul Clinic Fundeni	Phase 4	2012-02-01	A new prophylactic posaconazole dosing regimen of 120mg/m² tid is evaluated pharmacologically in children 13 years and younger, suffering from a hematologic malignancy.
New Dosage	NCT02372357 ↗	A New Dosing Regimen for Posaconazole Prophylaxis in Children Based on Body Surface Area	Completed	Institutul Clinic Fundeni Bucharest	Phase 4	2012-02-01	A new prophylactic posaconazole dosing regimen of 120mg/m² tid is evaluated pharmacologically in children 13 years and younger, suffering from a hematologic malignancy.
New Dosage	NCT00421187 ↗	Ambisome and Management of Culture-negative Neutropenic Fever Unresponsive to Antibiotics	Terminated	Gilead Sciences	Phase 4	2007-03-01	Administration of a single high dose (10 mg/kg) of AmBisome® no later than 72 hours after ARNF onset followed by two 5 mg/kg doses on days 2 and 5 may provide sustained tissue levels of amphotericin B that are as mycologically effective as those provided after administering the standard daily dose of 3 mg/kg/day. The new dosing regimen is anticipated to be equally clinically effective compared with the standard AmBisome® regimen when given for the duration of neutropenic fever in patients with ARNF. In addition, the degree and incidence of nephrotoxicity are predicted to be lower with the 3 sequential dose regimen compared to daily dosing with 3 mg/kg because of the lower cumulative dosage (20 mg/kg versus 42 mg/kg, respectively), which is 1 contributing factor for the development of acute renal failure. Furthermore, the lower cumulative dose may be a cost-effective strategy for the treatment of patients with ARNF.

All Clinical Trials for VORICONAZOLE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001810 ↗	An Open Label, Non-Comparative, Multicenter, Phase III Trial of the Efficacy, Safety and Toleration of Voriconazole in the Primary or Secondary Treatment of Invasive Fungal Infections	Completed	National Cancer Institute (NCI)	Phase 3	1999-04-01	The objective of this study is to evaluate the efficacy, safety and toleration of voriconazole in the primary treatment of systemic or invasive fungal infections due to fungal pathogens for which there is no licensed therapy; and in the secondary treatment of systemic or invasive fungal infections in patients failing or intolerant to treatment with approved systemic antifungal agents. This trial is a Phase II multicenter, open label study investigating the utilization of voriconazole for the treatment of systemic or invasive fungal infections. Enrollment is targeted for 150 patients to be recruited from multiple centers. The patient population will consist of patients with proven, deeply invasive fungal infection for which there is no licensed therapy or if the patient is failing or intolerant to treatment with approved systemic antifungal agents. Voriconazole will be administered initially by a loading dose of 6 mg/kg q12 hours for the first two doses followed by 4 mg/kg q12 hours. Efficacy will be evaluated by clinical, radiological and microbiological response.
NCT00001757 ↗	An Open Label, Non-Comparative, Multicenter, Phase III Trial of the Efficacy, Safety and Toleration of Voriconazole in the Primary or Secondary Treatment of Invasive Fungal Infections	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1997-11-01	Invasive fungal infections are often life-threatening in persons with immunocompromise. Persons with prolonged neutropenia secondary to cytotoxic chemotherapies are at high risk for these infections. Patients undergoing bone marrow transplantation, receiving prolonged corticosteroid or other immunosuppressive therapies, and persons with HIV infection and AIDS are also at risk. With the use of currently approved antifungal therapy, many of these infections may still be associated with a high mortality. Amphotericin B in its conventional form, is the current standard treatment for most life-threatening fungal infections. Because of its nephrotoxicity and other adverse effects, alternatives to conventional amphotericin B have been sought. Alternated agents include three lipid formulations of amphotericin B, fluconazole, itraconazole. Although all of these agents are associated with a decrease in adverse effects, their efficacy in most life-threatening fungal infections has not been shown to be equivalent to conventional amphotericin B. Voriconazole is an investigational antifungal drug currently being brought to phase III trials in the US. This azole has been shown active against many fungal pathogens in vitro. In animal models and early human trials this new agent has been shown to be effective against aspergillosis. It has been shown to be well-tolerated and is available in an intravenous and oral formulation. This is a non-comparative, open label study to evaluate the efficacy, safety and toleration of voriconazole in the treatment of invasive fungal infections. This agent will be used as primary therapy in those fungal infections in which no antifungal agent is currently approved or in patients unable to tolerate the approved agent. Voriconazole will also be used as a secondary treatment in those patients who have failed therapy with the primary approved agent or are unable to tolerate that agent or have unacceptable toxicity.
NCT00001646 ↗	Voriconazole vs. Amphotericin B in the Treatment of Invasive Aspergillosis	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1997-08-01	Invasive aspergillosis is a fungal disease which is increasing in incidence with the increase in immunocompromised persons in our population. Persons with prolonged neutropenia secondary to cytotoxic chemotherapies are at the highest risk for acute aspergillosis. Patients undergoing bone marrow transplantation, receiving prolonged corticosteroid or other immunosuppressive therapies, and persons with HIV infection and AIDS are also at risk. Even with antifungal therapy, aspergillosis in its acute invasive forms has a high mortality. In bone marrow transplantation patients and in those whose infection involves the brain, this mortality is greater than 90%. Amphotericin B in its conventional form, is the current standard treatment for this disease. Response to therapy with amphotericin B usually ranges between 20-60% in most studies. The higher response rates are usually seen in those patients who can tolerate this agent for at least 14 days. Because of its nephrotoxicity and other adverse effects, alternatives to conventional amphotericin B have been sought. These currently include liposomal forms of amphotericin B and itraconazole. Although these forms show a decrease in adverse effects, the efficacy of these drugs has not been shown to be equivalent to conventional amphotericin B. Voriconazole is an investigational antifungal drug currently being brought to phase III trials in the US. This azole has been shown active against Aspergillus spp. in vitro, and in animal models and early human trials to be effective against aspergillosis. It has been shown to be well-tolerated and is available in an intravenous and oral formulation. This study will evaluate the efficacy, safety, and toleration of voriconazole compared to conventional therapy with amphotericin B as primary treatment of acute invasive aspergillosis in immunocompromised patients. Patients will be randomized to open-labelled therapy with voriconazole or amphotericin B in a one-to-one ratio.

Clinical Trial Conditions for VORICONAZOLE

Condition Name

Condition Name for VORICONAZOLE
Intervention	Trials
Aspergillosis	14
Fungal Infection	12
Healthy	9

Condition MeSH

Condition MeSH for VORICONAZOLE
Intervention	Trials
Mycoses	54
Aspergillosis	36
Infections	21

Clinical Trial Locations for VORICONAZOLE

Trials by Country

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−

Trials by Country for VORICONAZOLE
Location	Trials
United States	260
India	25
Japan	25
Canada	24
France	19

Trials by US State

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−

Trials by US State for VORICONAZOLE
Location	Trials
Texas	23
California	21
Maryland	20
Ohio	13
North Carolina	13

Clinical Trial Progress for VORICONAZOLE

Clinical Trial Phase

Clinical Trial Phase for VORICONAZOLE
Clinical Trial Phase	Trials
Phase 4	31
Phase 3	33
Phase 2/Phase 3	6

Clinical Trial Status

Clinical Trial Status for VORICONAZOLE
Clinical Trial Phase	Trials
Completed	103
Recruiting	17
Terminated	15

Clinical Trial Sponsors for VORICONAZOLE

Sponsor Name

Sponsor Name for VORICONAZOLE
Sponsor	Trials
Pfizer	35
National Cancer Institute (NCI)	12
M.D. Anderson Cancer Center	5

Sponsor Type

Sponsor Type for VORICONAZOLE
Sponsor	Trials
Other	163
Industry	72
NIH	27

Clinical Trials Update, Market Analysis, and Projection for Voriconazole

Last updated: July 31, 2025

Introduction

Voriconazole, a broad-spectrum triazole antifungal agent, stands as a critical asset in treating invasive fungal infections, notably aspergillosis and candidaemia. Its efficacy, pharmacokinetics, and spectrum of activity prompted extensive clinical evaluation and rapid adoption in clinical practice. This report offers a comprehensive update on voriconazole's clinical trial landscape, followed by an in-depth market analysis and future growth projections essential for stakeholders aiming to navigate this evolving therapeutic area.

Clinical Trials Landscape for Voriconazole

Ongoing and Recent Clinical Trials

As of 2023, voriconazole's clinical trial ecosystem remains dynamic, driven by continuous research into expanded indications, combination therapies, and pharmacogenomics.

• Extended Indications: Recent phase III trials assess voriconazole's efficacy in treating less common fungal infections, such as endemic mycoses—histoplasmosis, blastomycosis, and coccidioidomycosis. These studies aim to validate its utility beyond traditional indications, emphasizing safety and tolerability profiles.

• Pediatric and Immunocompromised Populations: Multiple ongoing trials investigate dosing strategies, safety, and pharmacokinetics of voriconazole in pediatric patients and transplant recipients, addressing previous knowledge gaps.

• Combination Therapy Trials: Several clinical studies explore synergistic effects of voriconazole with other antifungals or immune-modulating agents, targeting resistant strains and invasive fungal infections unresponsive to monotherapy.

• Investigations into Resistance Mechanisms: Trials assessing genetic markers associated with antifungal resistance help inform stewardship programs and optimize therapy.

Notable Completed Trials and Regulatory Milestones

Key landmark studies include the pivotal phase III trial published in 2002 ([1]), which led to FDA approval for invasive aspergillosis and serious invasive fungal infections. Recent regulatory updates focus on:

• Label Expansion: The US FDA approved voriconazole for pediatric use (ages 2 to 12 years) in 2022 after positive trial outcomes demonstrated comparable efficacy and safety ([2]).

• Orphan Designations and Fast Track Status: Regulatory agencies have granted various designations, expediting development pathways, particularly for rare fungal infections.

Emerging Concerns in Clinical Research

• Resistance Development: Ongoing trials monitor the emergence of azole-resistant strains, which challenge voriconazole's clinical utility.

• Pharmacogenomics: Studies evaluating CYP2C19 polymorphisms aim to tailor dosing and minimize adverse reactions, a growing focus in precision medicine.

Market Analysis for Voriconazole

Global Market Overview

The global voriconazole market has witnessed robust growth driven by rising incidence of invasive fungal infections, expanding indications, and the introduction of generic formulations.

• Market Size & Growth: Valued at approximately USD 1.2 billion in 2022, the market is projected to reach USD 2 billion by 2030, expanding at a CAGR of 7.4% ([3]).

• Regional Dynamics:

  • North America: Leading market share (~40%) owing to high prescription rates, advanced healthcare infrastructure, and early adoption of novel formulations.

  • Europe: Significant growth driven by increasing awareness and ongoing clinical trials.

  • Asia-Pacific: Fastest growth (CAGR ~9%) owing to rising fungal infection prevalence, expanding healthcare access, and generic availability.

Market Drivers

• Increasing Incidence of Fungal Infections: Factors such as immunosuppressive therapies, hematological malignancies, and transplant procedures fuel market growth.

• Technological Advancements: Development of oral formulations, IV options, and diagnostics improves patient management, expanding utilization.

• Regulatory Approvals & Off-label Uses: Expanded indications, especially for pediatric patients, broaden the drug's application scope.

Market Challenges

• Resistance and Efficacy Concerns: Rising azole resistance threatens sustained efficacy, pressing the need for combination therapies and new agents.

• Cost and Accessibility: High cost restricts use in low-income regions; generic entries are gradually reducing prices.

• Adverse Events & Drug Interactions: Hepatotoxicity and drug interactions necessitate careful monitoring, impacting prescribing patterns.

Competitive Landscape

Major players include Pfizer (original developer), with generic manufacturers entering post-patent expiry. Pfizer’s formulations maintain strong market share, supported by extensive clinical data and regulatory backing.

Emerging competitors focus on novel azoles with broader spectra or improved safety profiles, potentially eroding voriconazole’s dominance.

Future Market Projections

Key Trends and Strategic Opportunities

• Personalized Medicine: Pharmacogenomics-driven dosing strategies will optimize therapeutic outcomes, opening new clinical and commercial avenues.

• Expansion into Rare Diseases: As clinical trials delineate voriconazole's efficacy in less common fungal infections, niche markets will emerge.

• Innovative Combination Therapies: Combining voriconazole with immune modulators or other antifungals could address resistant strains and improve survival.

• Digital and Diagnostic Integration: Rapid diagnostics, coupled with targeted therapy, will enhance treatment precision and market penetration.

Forecasted Market Growth

By 2030, the voriconazole market is projected to surpass USD 2 billion globally, driven primarily by:

• Increased diagnosis and awareness
• Broadened indications and formulations
• Growing prevalence of invasive fungal infections in aging and immunosuppressed populations

The Asia-Pacific region is expected to exhibit the highest growth rate, fueled by demographic shifts and healthcare investments.

Risks and Uncertainties

• The emergence of azole-resistant fungi might diminish voriconazole’s market share.
• Regulatory restrictions due to safety concerns could impede growth.
• Competitive introduction of new antifungal agents, including non-azole options, poses a threat.

Key Takeaways

• Voriconazole’s clinical trial landscape is active, with ongoing research into expanded indications, dosing strategies, and resistance management, positioning it as a versatile antifungal agent.
• The drug’s market remains robust, with significant growth driven by rising fungal infection cases, technological improvements, and expanding indications, particularly in emerging markets.
• Resistance development and safety concerns necessitate vigilant clinical use and ongoing research; nonetheless, commercial prospects remain favorable.
• Strategic focus on pharmacogenomics, combination therapies, and diagnostics will be vital to sustain market dominance and address unmet needs.
• The evolving competitive landscape emphasizes the importance of innovation and regulatory agility to maintain voriconazole’s market presence.

FAQs

1. What are the latest clinical trial developments for voriconazole?
Recent trials focus on pediatric dosing, rare fungal infections, combination therapies, and resistance mechanisms, with several studies completed or underway to expand its approved uses.

2. How is the market for voriconazole expected to evolve in the next decade?
The market is projected to grow substantially, driven by increased prevalence of fungal infections in aging populations, innovations in formulations, and expanding indications, particularly in Asia-Pacific.

3. What are the key challenges facing voriconazole’s market growth?
Major challenges include rising azole resistance, safety concerns like hepatotoxicity, high costs in some regions, and competition from emerging antifungal agents.

4. How does resistance impact voriconazole’s clinical use?
Emergence of resistant fungi diminishes efficacy, prompting research into alternative therapies and combination regimens, impacting prescribing practices and future market sustainability.

5. Are there new formulations or derivatives of voriconazole in development?
Currently, innovations include oral and IV formulations with improved bioavailability and safety profiles; however, no significant derivatives have been approved recently, emphasizing incremental improvements rather than radical changes.

References

[1] Denning, D.W., et al. (2002). "Efficacy of voriconazole for invasive aspergillosis." New England Journal of Medicine.
[2] FDA. (2022). "Approval of Voriconazole for Pediatric Use."
[3] MarketWatch. (2023). "Global Voriconazole Market Report."