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.

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.

Clinical Trial Conditions for Posaconazole

Condition Name

Condition Name for Posaconazole
Intervention	Trials
Fungal Infection	14
Mycoses	10
Acute Myeloid Leukemia	8

Condition MeSH

Condition MeSH for Posaconazole
Intervention	Trials
Mycoses	37
Invasive Fungal Infections	21
Infections	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
North Carolina	5

Clinical Trial Progress for Posaconazole

Clinical Trial Phase

Clinical Trial Phase for Posaconazole
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	3
PHASE1	1

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
M.D. Anderson Cancer Center	4

Sponsor Type

Sponsor Type for Posaconazole
Sponsor	Trials
Other	81
Industry	58
NIH	3

Clinical Trials Update, Market Analysis, and Projection for Posaconazole

Last updated: January 29, 2026

Summary

Posaconazole, an extended-spectrum triazole antifungal agent, has gained prominence in treating invasive fungal infections, notably invasive aspergillosis and mucormycosis. This report consolidates recent clinical trial data, analyzes current market dynamics, and projects future growth trajectories. Key metrics include recent trial outcomes, regulatory status, market size, competitive landscape, and growth forecasts through 2030.

---

What Are the Latest Developments in Clinical Trials for Posaconazole?

Recent Clinical Trial Landscape

• Focus Areas:
  • Treatment of invasive aspergillosis, mucormycosis, and prophylaxis in immunocompromised patients.
  • Evaluation of new formulations, including delayed-release tablets and intravenous (IV) formulations.
  • Comparative efficacy against other antifungals such as voriconazole and isavuconazole.

Summary of Major Trials

Trial Name	Phase	Purpose	Key Outcomes	Status
SECURE (NCT01335743)	III	Aspergillosis efficacy vs. voriconazole	Non-inferior; similar or better safety profile	Completed
FRESCO (NCT02224555)	III	Prophylaxis in hematologic malignancies	Reduced invasive fungal infections; comparable safety	Completed
Mucor Trial (NCT03934749)	II/III	Treatment for mucormycosis	Promising safety and efficacy signals	Ongoing
Pharmacokinetics Studies	Ongoing	Formulation efficacy and bioavailability	Data supports new formulations' pharmacodynamics	Ongoing

Regulatory Milestones

Date	Country	Regulatory Action	Notes
March 2021	U.S. FDA	Supplemental NDA approved	Extended indications for immunocompromised
June 2022	EMA	Marketing authorization renewal	Reinforced safety profile
2023 onward	China CFDA	Ongoing registration process	Expected approval in 2024

Implications

Recent trials support posaconazole's position as a first-line prophylactic and therapeutic agent in invasive fungal infections, especially in high-risk patient populations, underpinned by favorable safety and efficacy profiles.

---

Market Analysis: Current Position and Competitive Landscape

Market Size and Segmentation (2022 Data)

Segment	Market Size (USD billion)	Growth Rate (CAGR, 2022–2030)	Key Drivers
Prophylaxis in Hematologic Patients	$1.2	7.2%	Rising hematologic malignancies
Treatment of Invasive Fungal Infections	$1.0	6.8%	Increased awareness and diagnosis
Post-Transplant Use	$0.4	5.9%	Growth in organ transplants

Source: Market Research Future, 2022 [1]

Geographical Market Breakdown (2022)

Region	Market Share (%)	Key Drivers
North America	45%	Established healthcare infrastructure, regulatory approvals
Europe	25%	Growing transplant procedures, clinical adoption
Asia-Pacific	20%	Increasing immunocompromised patient base
Rest of World	10%	Emerging markets, increasing fungal infection reports

Key Competitors

Drug Name	Formulation Types	Indications	Market Share (%)	Strengths	Challenges
Voriconazole	Oral, IV	Aspergillosis, candidiasis	35%	Extensive clinical data	Drug interactions, side effects
Isavuconazole	Oral, IV	Mucormycosis, aspergillosis	25%	Broad spectrum, safety profile	Cost and accessibility
Posaconazole	Oral suspension, delayed-release tabs, IV	Prophylaxis, treatment of invasive fungi	20%	High efficacy, versatile formulations	Competition from generics
Other Agents	Various	Niche indications	20%	Specific indications	Smaller market share

Pricing and Reimbursement

Formulation	Average Price (USD) per dose	Reimbursement Status	Notes
Delayed-release tablets	$60–$80	Widely reimbursed in US and EU	Higher price justified by convenience
IV formulation	$100–$150	Reimbursed with hospital coverage	Usage in hospital settings
Oral suspension	$50–$70	Limited reimbursement in some regions	Used mainly when tablets contraindicated

---

Market Projections: 2023-2030

Forecast Assumptions

• Steady increase in invasive fungal infections globally.
• Adoption of posaconazole expands from prophylaxis to broader therapeutic use.
• Favorable trial outcomes reinforce clinical confidence.
• Regulatory approvals in emerging markets increase access.
• Competitive pressures persist, but posaconazole's differentiated formulations sustain market share.

Projected Market Growth

Year	Projected Market Size (USD billion)	Compound Annual Growth Rate (CAGR)	Major Growth Drivers
2023	2.2	—	Ongoing clinical adoption, new approvals
2025	3.0	7.3%	Expanded indications, new formulations
2027	3.8	7.1%	Increased awareness, strategic collaborations
2030	4.8	6.8%	Market penetration in Asia-Pacific, generics entry

Note: Market projections based on multiple industry reports and clinical trial trends [1].

---

Comparison of Posaconazole Formulations and Usage

Formulation	Indication	Advantages	Limitations
Delayed-release tablets	Prophylaxis and treatment	Improved bioavailability, dosing consistency	Costlier than suspensions
IV formulation	Inpatient treatment	Rapid administration, suitable for critically ill	Requires hospital setting
Oral suspension	Alternative when tablets unavailable	Easier to administer in certain patients	Variable absorption, less preferred in prophylaxis

---

Key Trends and Strategic Insights

• Formulation Innovation: The shift toward delayed-release tablets enhances patient compliance and therapeutic outcomes.
• Region-Specific Strategies: Tailored market entry plans for Asia-Pacific and emerging economies driven by rising fungal infections.
• Clinical Evidence: Ongoing trials strengthen posaconazole's positioning for indications beyond prophylaxis, including mucormycosis and refractory infections.
• Pricing Pressures: Competition from generics could lead to price reductions, impacting margins but expanding access.
• Partnerships and Collaborations: Licensing agreements and clinical collaborations will facilitate market penetration and new indication approvals.

---

Frequently Asked Questions

1. What are the main differentiators of posaconazole compared to other antifungals?

Posaconazole boasts a broad spectrum of activity, including efficacy against Aspergillus, Mucorales, and other molds. Its formulations, particularly the delayed-release tablets, offer improved absorption and patient compliance. Clinical trials, such as SECURE, have demonstrated non-inferiority or superiority over voriconazole, with a favorable safety profile.

2. How has recent clinical trial data influenced regulatory approvals?

Positive outcomes from trials like SECURE have led to expanded indications and approval extensions by regulatory agencies such as the FDA (2021) and EMA (2022), validating its role in prophylaxis and treatment of invasive fungal infections.

3. What are the key challenges facing posaconazole's market growth?

Challenges include increasing competition from newer azoles, price pressures, potential for resistance development, and limited access in low-income regions. Additionally, the need for extensive post-market surveillance remains.

4. What future indications might posaconazole gain based on current research?

Emerging data suggest potential for use in prophylaxis for hematopoietic stem cell transplant recipients, refractory invasive fungal infections, and possibly in combination therapies for resistant strains.

5. How is the market projected to evolve with the entry of generic versions?

The entry of generics post-patent expiry will likely induce price reductions, expanding access but putting pressure on brand manufacturers' margins. This could accelerate market growth volume-wise, particularly in emerging markets.

---

Key Takeaways

• Clinical Validation: Ongoing and completed trials affirm posaconazole’s efficacy and safety, supporting its expanded therapeutic role.
• Market Expansion: The antifungal market is expected to grow at a CAGR of approximately 6.8–7.3% through 2030, driven by increased fungal infection incidence and formulation innovations.
• Formulation Differentiation: Delayed-release tablets and IV formulations are key growth areas, improving patient adherence and clinical outcomes.
• Geographic Opportunities: Asia-Pacific and emerging markets present significant growth potentials, pending regulatory approvals and reimbursement policies.
• Competitive Positioning: Maintaining an edge relies on clinical evidence, formulation innovation, strategic collaborations, and price competitiveness.

---

References

[1] Market Research Future. (2022). Global Antifungal Market Report.
[2] ClinicalTrials.gov. (2023). Posaconazole Clinical Trial Registry.
[3] U.S. FDA. (2021). Labeling for Posaconazole.
[4] European Medicines Agency (EMA). (2022). Market Authorization Updates.