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	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.
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.

All Clinical Trials for VORICONAZOLE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
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.
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.
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.

Clinical Trial Conditions for VORICONAZOLE

Condition Name

Condition Name for VORICONAZOLE
Intervention	Trials
Aspergillosis	14
Fungal Infection	13
Healthy	9

Condition MeSH

Condition MeSH for VORICONAZOLE
Intervention	Trials
Mycoses	55
Aspergillosis	36
Infections	21

Clinical Trial Locations for VORICONAZOLE

Trials by Country

Trials by Country for VORICONAZOLE
Location	Trials
United States	262
India	25
Japan	25
Canada	24
France	21

Trials by US State

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

Clinical Trial Progress for VORICONAZOLE

Clinical Trial Phase

Clinical Trial Phase for VORICONAZOLE
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	4
PHASE1	1

Clinical Trial Status

Clinical Trial Status for VORICONAZOLE
Clinical Trial Phase	Trials
Completed	104
Recruiting	19
Terminated	15

Clinical Trial Sponsors for VORICONAZOLE

Sponsor Name

Sponsor Name for VORICONAZOLE
Sponsor	Trials
Pfizer	35
National Cancer Institute (NCI)	12
University of California, San Francisco	5

Sponsor Type

Sponsor Type for VORICONAZOLE
Sponsor	Trials
Other	165
Industry	76
NIH	27

Voriconazole: Clinical Trials Update, Market Analysis, and Projection

Last updated: April 24, 2026

What is voriconazole’s current clinical and regulatory status?

Voriconazole is an established systemic antifungal used for invasive aspergillosis, candidiasis (including fluconazole-resistant species), and other serious fungal infections. It is an approved small-molecule triazole antifungal, not a late-stage pipeline candidate in the way new chemical entities are.

Because voriconazole is already marketed, “clinical trials update” in practice is dominated by:

• New indications and comparative trials (often in specific patient subsets such as ICU populations, pediatric subsets, or refractory infections)
• Formulation and dosing optimization studies (therapeutic drug monitoring [TDM] practices, loading-dose regimens, pharmacokinetic [PK] refinements)
• Real-world evidence (RWE) studies evaluating tolerability, safety events (notably hepatotoxicity and QT effects), and regimen adherence
• Line extensions that affect market access (e.g., generic entries, biosimilar-like analogs are not relevant here, but generic brands do move pricing and tender dynamics)

Regulatory and label theme that continues to influence clinical practice: voriconazole dosing and exposure optimization are tightly linked to PK variability. Many contemporary studies and guidelines track TDM uptake and outcomes because under- or over-exposure increases failure risk or toxicity risk (e.g., visual disturbances, hepatotoxicity). FDA and major guideline bodies emphasize exposure-informed dosing for safety and efficacy. [1][2]

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What clinical-trials signals matter for business decisions in voriconazole?

Across ongoing and recently reported studies for voriconazole, three recurring “signal buckets” drive commercial and operational choices: safety management, exposure control, and regimen logistics.

1) Therapeutic drug monitoring adoption and outcomes

• Studies continue to evaluate whether routine TDM improves clinical outcomes versus symptom-guided dosing.
• Business impact: TDM changes cost of care and influences hospital formulary decisions, particularly in regions where lab turnaround time and clinician workflow make TDM harder.

2) Comparative performance versus alternative azoles and antifungals

• Trials and observational cohorts assess voriconazole against other azoles (posaconazole, isavuconazole) and against amphotericin B strategies in specific fungal ecologies or resistance contexts.
• Business impact: comparative effectiveness affects tender outcomes and switching behavior in hospitals and government formularies.

3) Safety event burden in real-world cohorts

• Hepatotoxicity, neurologic effects, photosensitivity/dermatologic events with longer exposure, QT-risk management, and drug-drug interaction monitoring are common endpoints in post-approval evidence.
• Business impact: safety burden changes use in high-comorbidity populations and impacts procurement in systems with strict pharmacovigilance protocols.

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What is voriconazole’s market structure and competitive landscape?

Voriconazole is a mature antifungal with significant penetration through generics in many markets. The commercial landscape is primarily shaped by:

• Brand-to-generic substitution at patent expiry
• Hospital tendering cycles and national procurement rules
• Formulary protocols that may require IV-to-oral step-down and TDM integration
• Competition from newer azoles (posaconazole, isavuconazole) in aspergillosis pathways and prophylaxis algorithms
• Safety and interaction profile management, which can increase monitoring costs even when drug acquisition price falls

Competitive “practical substitutes” by indication pathway

• Invasive aspergillosis: voriconazole is a core comparator to posaconazole and isavuconazole strategies in guidelines and real-world practice. [2]
• Candidiasis and step-down therapy: selection depends on species distribution, resistance patterns, liver function, and interaction burden.
• Prophylaxis and high-risk hematology/ICU populations: newer prophylaxis algorithms often reduce voriconazole share relative to other azoles depending on local guidance and reimbursement.

Market power drivers

• Tender price compression: generic availability typically drives lower net price and increased price competition.
• Protocol stickiness: once hospitals adopt an “aspergillosis pathway” or prophylaxis algorithm, switching costs are mainly operational (TDM workflow, dosing protocols, PK monitoring capabilities).
• Drug-drug interaction burden: CYP-mediated interactions influence prescribing behavior and can shift use away from settings with complex polypharmacy, even if drug price is low.

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What do pricing and utilization trends imply for forward demand?

With a mature asset, forward demand is less about “new-to-market” adoption and more about share movements and intensity of use within established fungal care pathways.

Demand tailwinds

• Ongoing incidence of invasive fungal disease in oncology and transplant populations sustains baseline demand for systemic antifungals.
• Clinical protocol refinement (dose optimization and monitoring) can reduce treatment discontinuation and support continued use of voriconazole where it remains preferred.

Demand headwinds

• Share erosion from newer azoles in some geographies and indications based on tolerability, dosing convenience, and prophylaxis pathway alignment.
• Safety monitoring and interaction management requirements can reduce “default use” in high-turnover, low-monitoring environments.

Net effect for business planning

• Market growth is usually modest and driven more by volume stability and population-level incidence changes than by margin expansion.
• Pricing erosion from generic competition is the dominant financial force in many countries, with offset from volume retention where protocols keep voriconazole in first-line sequences.

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How big is the opportunity going forward and what is the market projection?

A precise numeric market projection (global $ and CAGR) requires current market sizing datasets and time-bounded forecast models (e.g., IQVIA/Datamonitor style series). Those figures are not provided in the cited materials below. Therefore, a projection here is expressed as a directional forecast framework tied to identifiable drivers rather than unreferenced dollar forecasts.

3-scenario projection (directional)

Scenario A: Volume-stable, price-down

• Generics maintain broad access.
• Net market value declines or stagnates due to tender-driven price pressure.
• Primary growth is volume in higher-risk populations with minimal switching away from voriconazole.

Scenario B: Moderate share loss to newer azoles

• Hospitals and payers shift aspergillosis and prophylaxis algorithms toward competing azoles.
• Net value declines faster than volume growth.
• TDM complexity favors competitors that reduce monitoring intensity.

Scenario C: Protocol-driven retention

• Continued guideline-consistent use and strong TDM outcomes support voriconazole retention.
• Share remains stable in settings with robust monitoring infrastructure.
• Net value stabilizes relative to Scenario B.

This directional projection aligns with how antifungal formularies typically evolve in mature, generic-exposed markets while new azoles gain share through algorithm changes. [2]

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Where do clinical and economic endpoints align with payer and hospital decision-making?

Voriconazole’s continued use depends on the intersection of clinical outcomes and operating metrics.

Decision endpoints used in practice

• Time-to-appropriate therapy in suspected invasive fungal infection
• Overall survival and response at defined treatment timepoints
• Discontinuation rates due to toxicity
• TDM feasibility metrics (sample-to-result turnaround, protocol adherence)
• Management effectiveness for drug-drug interactions

Economic endpoints that drive procurement

• Net acquisition cost after tender and rebate structure
• Cost of monitoring (TDM assays, liver enzyme and ECG monitoring)
• Cost of adverse event management (hepatotoxicity, neuro-ocular events, photosensitivity outcomes)
• Treatment duration distribution and step-down success rates

Guideline emphasis on dosing and therapeutic monitoring links directly to these operational and cost endpoints. [1][2]

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Key implications for R&D and investment (even though the asset is mature)

Even for a mature drug, business decisions still hinge on where incremental value can be captured.

1) Formulation and administration improvements

• Better IV presentation, stability improvements, and IV-to-oral transition support can reduce nursing time, infusion delays, and wastage.
• These changes can create differentiation in tender specifications even when active ingredient is the same.

2) TDM workflow and decision support

• Any product-integrated support that improves adherence to exposure targets can reduce failure and toxicity rates and protect formulary position.
• This is operational R&D rather than new molecular discovery.

3) Evidence-generation focused on real-world implementation

• RWE studies that show improved safety outcomes with structured monitoring can support contracting and formulary retention.
• Outcomes that matter to hospital administrators include discontinuation rates and length-of-stay impact.

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Key Takeaways

• Voriconazole is a mature systemic antifungal where “clinical trials update” centers on TDM adoption, dosing optimization, comparative performance against alternative azoles, and real-world safety event management. [1][2]
• The market is structurally defined by generic penetration, hospital tendering, and protocol-based switching behavior rather than by new chemical entity launches.
• Forward value growth is likely modest and constrained by price compression; directional outcomes depend on whether newer azoles gain share through guideline algorithm changes versus whether monitoring-enabled voriconazole pathways retain first-line status. [2]

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FAQs

1) Is voriconazole still used as a first-line therapy?

Yes. It remains a core systemic option in invasive aspergillosis pathways in guideline-consistent care, with ongoing use supported by dosing and monitoring protocols. [2]

2) Why does therapeutic drug monitoring keep appearing in voriconazole studies?

Voriconazole has high PK variability and toxicity risk that correlate with exposure; TDM supports dose individualization to balance efficacy and safety. [1][2]

3) What are the main safety concerns that influence utilization?

Hepatotoxicity and other adverse effects linked to exposure and drug interactions, alongside monitoring needs such as liver function and ECG assessment, shape prescribing and discontinuation rates. [1][2]

4) What most strongly drives market share versus newer azoles?

Hospital prophylaxis and invasive aspergillosis treatment algorithms, local formulary rules, and the operational ability to implement dosing protocols and monitoring influence switching. [2]

5) Does generic competition limit market growth?

In most markets, generic entries compress pricing and drive value trends more than volume growth, so net market expansion is typically limited unless share is retained through protocol fit and operational outcomes.

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References

[1] U.S. Food and Drug Administration. (2019). Vfend (voriconazole) prescribing information. FDA.
[2] Cornely, O. A., Alastruey-Izquierdo, A., Arenz, D., et al. (2017). Guideline for the diagnosis and management of aspergillosis from the European Confederation of Medical Mycology (ECMM) and the Global Action Fund for Fungal Infections (GAFFI). The Lancet Infectious Diseases.