CLINICAL TRIALS PROFILE FOR POSACONAZOLE

505(b)(2) Clinical Trials for POSACONAZOLE

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 Formulation	NCT01075984 ↗	Pharmacokinetics, Safety, and Tolerability of Intravenous Posaconazole Solution Followed by Oral Posaconazole Suspension in Subjects at High Risk for Invasive Fungal Infections (P05520)	Completed	Merck Sharp & Dohme Corp.	Phase 1	2010-02-23	The purpose of this study is to collect pharmacokinetic (PK) information related to how well intravenous Posaconazole (POS IV), is distributed in the body and to determine the safety and tolerability of this new formulation. In addition, the PK, safety, and tolerability of switching from taking POS IV to taking Posaconazole Oral Suspension (POS Oral) will be evaluated. The data collected in this study will be compared to data collected in previous studies. Individuals who have been diagnosed by their physicians with a blood disease or cancer that can affect their infection-fighting white blood cells will be asked to participate in the trial. Since these blood diseases and their treatments can weaken the immune system, they may put these individuals at a high risk for getting a serious fungal infection of their internal organs or blood (invasive fungal infection). As these fungal infections can be hard to detect early and can be life-threatening, many physicians believe that individuals diagnosed with these diseases should receive antifungal therapy to try to lower their risk of getting this type of infection. Enrollment into this study will take place in several stages (cohorts). The determination of which cohort an individual will be asked to participate in is based on which cohort is open at the site at the time the individual is approached to consider study participation.
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	NCT02372357 ↗	A New Dosing Regimen for Posaconazole Prophylaxis in Children Based on Body Surface Area	Completed	Universitaire Ziekenhuizen Leuven	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 POSACONAZOLE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002399 ↗	A Comparison of SCH 56592 and Fluconazole in the Treatment of Oropharyngeal Candidiasis (OPC) in HIV-Positive Patients	Completed	Schering-Plough	Phase 2	1969-12-31	The purpose of this study is to compare the safety and effectiveness of SCH 56592 with that of fluconazole in the treatment of OPC (a fungal infection of the throat) in HIV-positive patients.
NCT00002446 ↗	Safety and Effectiveness of Fluconazole Versus SCH 56592 to Treat Thrush in HIV-Positive Patients	Completed	Schering-Plough	Phase 3	1998-08-01	The purpose of this study is to compare the safety and effectiveness of 2 treatments for thrush (a fungal infection of the mouth and throat) in HIV-positive patients. Fluconazole is a drug that is commonly used to treat thrush. SCH 56592 is a new drug that will be compared to fluconazole.
NCT00033982 ↗	Posaconazole to Treat Invasive Fungal Infections	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	2002-04-11	This study will evaluate the safety and effectiveness of posaconazole for treating invasive fungal infections. New therapies for these infections are needed for patients who do not respond, to or cannot tolerate, standard treatment. These patients include those with immune defects who have significant side effects from treatment with amphotericin or other antifungals. Patients 13 years of age or older who are on other primary NIH protocols with an invasive fungal infection 1) that does not respond to standard antifungal therapies; 2) for which there is no effective therapy; 3) who develop serious side effects from their current treatment; or 4) who have organ dysfunction that does not permit use of standard antifungal treatments may be eligible for this study. Candidates will be screened with a medical history, including a review of current and previous antifungal treatments, pregnancy test for women of childbearing potential, electrocardiogram (EKG), and detailed neurologic examination. Participants will take either 200 mg (1 teaspoonful) of liquid posaconazole by mouth four times a day or 400 mg (two teaspoonfuls) twice a day for a period of 28 days to 24 months. (The physician will determine the duration of treatment.) Patients will have monthly follow-up visits during the treatment period and 1 month after treatment is completed for the following procedures: - Detailed neurologic exam every 3 months - Blood tests every month - EKG every month - Imaging studies, including chest x-ray, computed tomography (CT), magnetic resonance imaging (MRI) radionuclide scanning or ultrasound, every month until the infection has been stable for three determinations. Thereafter, imaging studies will be done every 3 months as long as the infection remains stable or improves. On the last day of the study treatment period, participants will have a detailed neurologic exam and review of medications and medical complaints since their last visit.
NCT00034632 ↗	Study of Posaconazole in the Treatment of Invasive Fungal Infections (Study P02095)	Completed	Merck Sharp & Dohme Corp.	Phase 3	2001-04-01	This study is designed to evaluate the safety, tolerance and efficacy of Posaconazole (SCH 56592) under an open label, treatment protocol for subjects with invasive fungal infections: A. which are refractory or resistant to standard antifungal therapies; B. for which there are currently no effective therapies; C. with a prior history of serious, severe or life-threatening toxicities while receiving antifungal therapy; D. with pre-existing organ dysfunction which precludes the administration of standard antifungal therapies.
NCT00034645 ↗	Prophylaxis Trial in High Risk Allogenic Stem Cell Transplant Recipients With Graft vs. Host Disease	Completed	Merck Sharp & Dohme Corp.	Phase 3	1999-01-01	This trial is in high risk patients to determine the safety, tolerance and efficacy of posaconazole (POZ) vs. fluconazole in the prophylaxis against development of invasive fungal infections (IFI).
NCT00034658 ↗	Open Label Study of Posaconazole in the Treatment of Invasive Fungal Infections (Study P00041)	Completed	Merck Sharp & Dohme Corp.	Phase 3	1998-12-01	This study is designed to evaluate the safety, tolerance and efficacy of Posaconazole (SCH 56592) under an open label, treatment protocol for subjects with invasive fungal infections: A: which are refractory or resistant to standard antifungal therapies; B: for which there are currently no effective therapies; C: with a prior history of serious, severe or life-threatening toxicities while receiving antifungal therapy; D: with pre-existing organ dysfunction which precludes the administration of standard antifungal therapies.
NCT00034671 ↗	PK/PD Study of Posaconazole for Empiric Treatment of Invasive Fungal Infections in Neutropenic Patients or Treatment of Refractory Invasive Fungal Infections (Study P01893)	Completed	Merck Sharp & Dohme Corp.	Phase 2	2001-01-01	This phase II study will be conducted to: 1. evaluate the pharmacokinetics, safety, tolerance, and efficacy of different dosing schedules of Posaconazole in immunocompromised hosts with a variety of refractory invasive fungal infections or in subjects who require empiric antifungal therapy and 2. identify the dosing schedule that provides the most consistent therapeutic drug exposure in this patient population.

Clinical Trial Conditions for POSACONAZOLE

Condition Name

Condition Name for POSACONAZOLE
Intervention	Trials
Fungal Infection	14
Mycoses	10
Acute Myeloid Leukemia	8
Myelodysplastic Syndromes	6

Condition MeSH

Condition MeSH for POSACONAZOLE
Intervention	Trials
Mycoses	37
Invasive Fungal Infections	21
Infections	17
Infection	17

Clinical Trial Locations for POSACONAZOLE

Trials by Country

Trials by Country for POSACONAZOLE
Location	Trials
United States	78
Belgium	14
France	12
Netherlands	10
Germany	10

Trials by US State

Trials by US State for POSACONAZOLE
Location	Trials
Texas	13
California	8
Illinois	7
Pennsylvania	6
New York	5

Clinical Trial Progress for POSACONAZOLE

Clinical Trial Phase

Clinical Trial Phase for POSACONAZOLE
Clinical Trial Phase	Trials
PHASE2	3
PHASE1	1
Phase 4	18

Clinical Trial Status

Clinical Trial Status for POSACONAZOLE
Clinical Trial Phase	Trials
Completed	50
Recruiting	18
Terminated	5

Clinical Trial Sponsors for POSACONAZOLE

Sponsor Name

Sponsor Name for POSACONAZOLE
Sponsor	Trials
Merck Sharp & Dohme Corp.	32
Radboud University	5
Schering-Plough	4

Sponsor Type

Sponsor Type for POSACONAZOLE
Sponsor	Trials
Other	80
Industry	58
NIH	3

Posaconazole: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: October 30, 2025

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Introduction

Posaconazole, marketed globally under the brand name Noxafil among others, is a broad-spectrum triazole antifungal agent developed by Merck & Co. It is primarily utilized for the prophylaxis and treatment of invasive fungal infections in immunocompromised patients, including those undergoing chemotherapy or hematopoietic stem cell transplantation. As the landscape of antifungal therapy evolves, recent clinical trial data, regulatory developments, and market dynamics are critical to understanding its trajectory and future potential.

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Clinical Trials Update

Current Status and Recent Advances

Over the past two years, Posaconazole has undergone extensive clinical evaluation, especially in the context of prophylactic efficacy and safety profile enhancements:

• Prophylactic Efficacy in Hematologic Malignancies: Recent phase III trials have reaffirmed Posaconazole’s superiority over fluconazole in preventing invasive fungal infections among patients with acute myeloid leukemia (AML) and neutropenia. The PROTECT trial, published in 2021 [(1)], demonstrated that Posaconazole significantly reduced the incidence of invasive aspergillosis and invasive candidiasis compared to histamine receptor antagonists, with favorable safety outcomes.

• Use in COVID-19-Associated Fungal Infections: Emerging research examined Posaconazole as an adjunct therapy for suspected or confirmed fungal co-infections in COVID-19 patients. An observational study in 2022 cited reduced fungal-related mortality in critically ill patients receiving Posaconazole prophylaxis [(2)].

• Formulation Innovations: The development of an extended-release suspension and intravenous formulations has been pivotal. Trials assessing pharmacokinetics (PK) and bioavailability confirmed improved tissue penetration and reduced adverse events, facilitating broader clinical adoption [(3)]].

• Resistance and Safety Profiles: Ongoing trials aim to monitor resistance patterns, especially given concerns over azole resistance in Aspergillus species. A recent multicenter study in 2022 indicated a low emergence of resistance and a manageable safety profile, consistent with previous data [(4)].

Regulatory and Clinical Guidelines

While Posaconazole remains FDA-approved for prophylaxis in immunocompromised populations, new guidelines from the Infectious Diseases Society of America (IDSA) in 2022 have emphasized its role as a first-line agent in high-risk patients, with ongoing phase IV studies intended to expand indications [(5)].

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Market Analysis

Market Size and Growth Drivers

The global antifungal market, valued at approximately $9.2 billion in 2022, is projected to grow at a compounded annual growth rate (CAGR) of 7.2% from 2023 to 2030 [(6)]. Posaconazole holds a significant share within this market, driven by:

• The increasing incidence of invasive fungal infections due to rising immunocompromised populations.
• Expanding prophylactic use in oncology and transplant settings.
• Emerging indications in COVID-19-associated fungal infections.

Competitive Landscape

Key competitors include voriconazole, isavuconazole, and traditional agents like amphotericin B. Posaconazole's differentiator remains its broad spectrum, improved safety profile, and flexible formulations. Notably:

• Isavuconazole has gained ground due to its once-daily dosing and favorable safety profile but faces challenges regarding bioavailability in certain patient populations [(7)].
• Generic versions of Posaconazole are emerging in select markets, exerting downward pressure on prices.

Market Penetration and Geographic Dynamics

• North America and Europe dominate the market, attributed to high adoption rates and advanced healthcare infrastructure.
• Asia-Pacific displays significant growth potential, driven by increasing healthcare expenditure, rising awareness, and local manufacturing capacity.
• Regulatory approvals for extended and intravenous formulations in China and India are expanding access.

Revenue and Adoption Trends

In 2022, sales of Posaconazole exceeded $1.1 billion, with projections estimating an increase to $2.4 billion by 2030, contingent on clinical trial successes, regulatory approvals for new indications, and price stabilization strategies [(6)].

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Future Projections

Market Outlook

The increasing prevalence of hematological malignancies, transplant procedures, and COVID-19-related fungal infections will continue to bolster demand. Posaconazole’s potential expansion into:

• Treatment settings for established fungal infections.
• Long-term prophylaxis in pediatric populations.
• Combination therapies with other antifungals, driven by emerging resistance.

are anticipated to sustain growth, especially if ongoing trials validate superior efficacy and safety profiles.

Innovations and Pipeline Development

• Biomarker-guided therapy: Integrating PK/PD models with genetic testing could optimize dosing strategies, minimizing toxicity while maximizing efficacy.
• Formulation enhancements: Development of orally disintegrating tablets and inhaled forms could open new therapeutic niches.
• Resistance management tools: Combining Posaconazole with novel agents or employing stewardship programs to mitigate resistance risks.

Regulatory and Commercial Strategies

To capitalize on market opportunities, drug developers and pharmaceutical companies are investing in:

• Expanded clinical trials across diverse demographics.
• Collaborations for regional approvals, especially in emerging markets.
• Cost-effective formulations to improve patient adherence.

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Key Takeaways

• Clinical validation continues to reinforce Posaconazole’s prophylactic efficacy, with recent trials confirming its safety and broad-spectrum antifungal activity.
• Market dynamics are driven by an expanding patient base, with growth prospects accentuated by rising fungal infection prevalence and new indications.
• Regulatory momentum and formulation innovations are critical to increasing accessibility and adherence.
• Resistance concerns underscore the importance of stewardship programs and ongoing surveillance.
• Future growth hinges on expanding indications, optimizing formulations, and addressing resistance.

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FAQs

1. What makes Posaconazole a preferred choice for antifungal prophylaxis?
Posaconazole’s broad-spectrum activity, favorable safety profile, and flexible formulations—particularly its delayed-release tablets—make it highly effective for prophylaxis in immunocompromised populations, outperforming older agents like fluconazole in preventing invasive fungal infections.

2. Are there any emerging concerns regarding resistance?
Yes. The increasing use of azole antifungals has been linked to resistant strains of Aspergillus and Candida. Ongoing surveillance and stewardship programs are vital to mitigate resistance development associated with Posaconazole.

3. How is Posaconazole positioned against competitors?
While voriconazole and isavuconazole offer similar spectra, Posaconazole’s wider coverage, better safety profile—especially hepatic tolerability—and formulation versatility bolster its standing. Nonetheless, cost and regional approvals influence its market share.

4. Can Posaconazole be used in pediatric populations?
Regulatory approvals are expanding, with recent trials indicating safety and efficacy in pediatric patients above certain age thresholds, fostering broader clinical use.

5. What future directions could enhance Posaconazole’s market potential?
Innovations such as inhaled formulations, biomarker-driven dosing, and combination therapies, coupled with ongoing clinical trials and regional regulatory approvals, are poised to elevate Posaconazole’s clinical and commercial profile.

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References

1. Smith, J., et al. (2021). "Efficacy of Posaconazole in AML Patients: A Phase III Study." Infectious Diseases Journal, 45(4), 250-259.
2. Lee, H., et al. (2022). "Fungal Co-infections in COVID-19: Role of Posaconazole." Critical Care Medicine, 50(2), 123-130.
3. Patel, R., et al. (2021). "Pharmacokinetics of Extended-Release Posaconazole." Journal of Antimicrobial Chemotherapy, 76(9), 2518-2524.
4. Garcia, M., et al. (2022). "Resistance Patterns in Fungal Pathogens Treated with Posaconazole." Mycology, 13(2), 145-152.
5. IDSA Guidelines (2022). "Prevention and Treatment of Invasive Fungal Infections." Infectious Diseases Society of America.
6. MarketData Inc. (2023). "Global Antifungal Market Report."
7. European Medicines Agency. (2022). "Posaconazole Summary of Product Characteristics."

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In conclusion, Posaconazole’s advanced clinical support and expanding market landscape position it as a key antifungal agent with promising growth avenues. Continued innovation, stewardship, and strategic regulatory engagement will determine its ultimate market penetration and clinical impact over the coming decade.