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
University of California, San Francisco	5

Sponsor Type

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

Voriconazole: Clinical Trials Update, Market Analysis, and Projections

Introduction to Voriconazole

Voriconazole is a third-generation triazole antifungal drug widely used for treating invasive fungal infections, particularly those caused by Aspergillus and Candida species. It is a critical treatment option for patients with weakened immune systems.

Clinical Trials Update

TFF VORI Clinical Program

TFF Pharmaceuticals has been conducting clinical trials for its Voriconazole Inhalation Powder (TFF VORI), aimed at treating invasive pulmonary aspergillosis (IPA). Here are the key updates:

• Phase 2 and Expanded Access Program (EAP): The Phase 2 trial compared TFF VORI with oral voriconazole in a 3:1 randomization. As of March 8, 2024, nine patients have been treated with TFF VORI, with updated safety data showing a favorable safety and tolerability profile. The efficacy data indicate antifungal activity, and the company has decided to focus resources on next steps for the program, including potential Phase 3 development[1].

• Patient Outcomes: Five of the seven initial patients treated with TFF VORI completed at least eight weeks of therapy, and one additional patient from the Phase 2 study completed treatment, bringing the total number of patients assessed for efficacy to six. The data suggest that TFF VORI could offer a promising alternative to oral voriconazole[1].

VORIGENIPHARM Study

The VORIGENIPHARM study, funded by the Spanish Health Research and Development Strategy, is a Phase IV pragmatic, multicentre, randomised, single-blind trial. This study aims to evaluate the efficiency of pre-emptive voriconazole genotyping compared to routine practice:

• Genotyping and Dosing: Patients are genotyped for CYP2C19 alleles and randomized to receive voriconazole doses based on a pharmacogenetic algorithm or according to clinical practice guidelines. The primary outcome is achieving serum voriconazole levels within the therapeutic range on the fifth day of treatment[3].

• Clinical Implications: The study highlights the importance of pharmacogenetic dosing to optimize voriconazole levels, reduce adverse effects, and improve treatment outcomes. Achieving therapeutic levels early in treatment is crucial for the prognosis of fungal infections and the tolerability of the treatment[3].

Market Analysis

Current Market Size and Growth

The voriconazole market is significant and growing:

• Market Size: The global voriconazole market was valued at USD 16.36 billion in 2023 and is projected to reach USD 20.53 billion by 2030, growing at a CAGR of 3.9% during the forecast period[5].

• Key Drivers: The market growth is driven by rising awareness of fungal infections, advancements in antifungal therapeutics, and increasing investments in research and development. Emerging economies with improved healthcare infrastructure also present substantial opportunities[2][5].

Market Dynamics

• Applications and End Users: Voriconazole is used in various settings, including clinics, homecare, and hospitals. The drug is available in different formulations such as injections, oral suspensions, and tablets, catering to different patient demographics and therapeutic areas[2].

• Competitive Landscape: The market faces competitive pressures from generic drugs and newer antifungal agents. However, voriconazole remains a first-line treatment for invasive fungal diseases due to its broad-spectrum activity and established efficacy[2][4].

Challenges and Opportunities

• Side Effects and Resistance: Despite its efficacy, voriconazole has potential side effects such as hepatotoxicity and neurotoxicity. Additionally, rising instances of antifungal resistance and the cost burden of long-term treatment pose challenges to market growth[2].

• Regulatory and Economic Factors: Stringent regulatory approvals and government initiatives for antifungal drug development influence the market. The cost-effectiveness of pre-emptive genotyping strategies, as explored in the VORIGENIPHARM study, could also impact market dynamics[3][5].

Projections and Future Outlook

Market Growth Projections

• Forecast Period: The voriconazole market is expected to continue growing, driven by increasing demand for effective antifungal treatments. The projected CAGR of 3.9% from 2024 to 2030 indicates a steady and significant expansion[5].

• Emerging Markets: Emerging economies with improving healthcare infrastructure are expected to contribute significantly to market growth. Government subsidies and initiatives for antifungal drug development will further support this expansion[2][5].

Innovations and Research

• Inhalation Formulations: Innovations like TFF VORI, which is in clinical development, offer potential alternatives to traditional oral and intravenous formulations. These new formulations could address unmet needs in specific patient populations, such as those with lung transplant or IPA[1].

• Pharmacogenetics: The integration of pharmacogenetic strategies, as seen in the VORIGENIPHARM study, is expected to enhance the efficacy and safety of voriconazole treatment. This approach could become more widespread, improving patient outcomes and reducing adverse effects[3].

Key Takeaways

• Clinical Trials: TFF VORI and the VORIGENIPHARM study highlight ongoing efforts to improve voriconazole treatment outcomes through innovative formulations and pharmacogenetic dosing.
• Market Growth: The voriconazole market is projected to grow at a CAGR of 3.9% from 2024 to 2030, driven by increasing demand and advancements in antifungal therapeutics.
• Challenges and Opportunities: Despite challenges such as side effects and antifungal resistance, the market is expected to benefit from emerging economies, government initiatives, and innovative research.

FAQs

What is the current status of TFF VORI in clinical trials?

TFF VORI is in the Phase 2 trial stage, with updated data showing a favorable safety and tolerability profile and antifungal activity. The company has decided to focus resources on next steps for the program, including potential Phase 3 development[1].

How does the VORIGENIPHARM study impact voriconazole treatment?

The VORIGENIPHARM study evaluates the efficiency of pre-emptive voriconazole genotyping, which can optimize serum voriconazole levels, reduce adverse effects, and improve treatment outcomes. This approach could become a standard practice in clinical settings[3].

What are the key drivers of the voriconazole market growth?

The market growth is driven by rising awareness of fungal infections, advancements in antifungal therapeutics, increasing investments in research and development, and emerging economies with improved healthcare infrastructure[2][5].

What are the challenges facing the voriconazole market?

Challenges include potential side effects such as hepatotoxicity and neurotoxicity, rising instances of antifungal resistance, and the cost burden of long-term treatment. Stringent regulatory approvals and competition from generic drugs also pose challenges[2].

What is the projected market size of voriconazole by 2030?

The global voriconazole market is projected to reach USD 20.53 billion by 2030, growing at a CAGR of 3.9% during the forecast period[5].

Sources

1. TFF Pharmaceuticals Announces Updated Data from the Tacrolimus Inhalation Powder and Voriconazole Inhalation Powder Clinical Programs. Biospace, March 27, 2024.
2. Oral Voriconazole Market Size & Share 2025-2030. 360iResearch.
3. VORIGENIPHARM study protocol. BMJ Open, October 2020.
4. VFEND® (voriconazole) Clinical Studies. Pfizer Medical Information.
5. Voriconazole Market Trends & Growth Insights 2024-2030. Verified Market Reports.