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
New York	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

POSACONAZOLE Market Analysis and Financial Projection

Last updated: April 30, 2026

Posaconazole: Clinical Trials Update and Market Forecast

What is posaconazole and where does it sit in the clinical pipeline?

Posaconazole is a broad-spectrum triazole antifungal used in invasive fungal disease (IFD), including invasive aspergillosis and invasive candidiasis, with formulations optimized for oral and suspension exposure characteristics. The current commercial landscape is dominated by established indications rather than a large set of late-stage, clearly differentiated new-drug programs under the same active ingredient name.

Clinical development reality for “posaconazole” (new trials) Public-facing clinical activity under the brand/INN “posaconazole” is largely characterized by:

• formulation refinements, dosing optimization, and special-population studies;
• comparative studies versus other antifungals for specific IFD subtypes or salvage settings;
• registry-style or post-authorization studies tied to safety and effectiveness in routine care.

Implication for pipeline assessment For market projection, posaconazole’s value proposition is anchored to:

• ongoing demand for IFD treatment and prophylaxis in high-risk groups (hematology and transplant);
• guideline-consistent use where posaconazole is preferred due to oral bioavailability (with modern formulations);
• continuity risk that is more driven by competitor class dynamics (new triazoles, combination strategies) than by rapid pipeline replacement.

---

What are the key clinical trial endpoints and typical design patterns?

Across ongoing and recently reported posaconazole studies, the measurable endpoints cluster around standard IFD frameworks:

Clinical domain	Common endpoints	Typical comparator patterns
Invasive aspergillosis	global response (complete/partial), survival, microbiological clearance	voriconazole, amphotericin B formulations
Invasive candidiasis	response and survival by baseline risk, safety	echinocandins, amphotericin B
IFD prophylaxis	incidence of breakthrough fungal infection, safety/tolerability	fluconazole, voriconazole, other prophylaxis regimens
Salvage/refractory IFD	response in refractory populations, time-to-treatment failure	alternative antifungals depending on region and standard of care

Operational interpretation Most “new trial” activity does not change the approved therapeutic positioning in a step-function way; it tends to maintain use through evidence generation that supports guideline inclusion and payer coverage.

---

Market analysis: demand drivers, pricing mechanics, and competitive set

How big is the posaconazole market and what drives volumes?

The market is driven by incidence and risk stratification in:

• acute myeloid leukemia (AML) and other hematologic malignancies;
• stem cell transplant (SCT) settings;
• immunosuppressed populations receiving corticosteroids and chemotherapy;
• oncology supportive-care pathways where prophylaxis reduces breakthrough IFD.

Primary demand levers

1. Prophylaxis adoption in high-risk cohorts (where oral regimens reduce IV logistics).
2. Shift in regimen choice toward oral triazoles where bioavailability supports therapeutic exposure.
3. Hospital formulary tightening that favors cost-effective antifungal stewardship, but maintains coverage for guideline-standard agents.

---

Who competes with posaconazole and how does that affect share?

The competitive set is dominated by other antifungals that overlap in indications and prophylaxis strategy:

Competitive class	Examples	Competitive mechanism vs posaconazole
Other triazoles	voriconazole, isavuconazole	oral step therapy, differing safety profiles, payer preference
Echinocandins	caspofungin, micafungin, anidulafungin	candidiasis targeted use, prophylaxis and initial therapy switching
Amphotericin B formulations	liposomal amphotericin B	efficacy in severe IFD, but IV and tolerability burden
Newer agents	investigational pipeline across class	long-term share risk if approvals broaden into key prophylaxis/therapy niches

Net effect on projection For posaconazole, market growth is less about “indication expansion breakthroughs” and more about sustaining prophylaxis use while managing substitution risk from isavuconazole and newer class entrants.

---

What are the market structure characteristics that influence pricing and revenue?

Key commercial mechanics affecting revenue:

• Segment mix: prophylaxis versus treatment; prophylaxis is volume-heavy but often price-constrained.
• Site of care: inpatient oncology/transplant centers carry formularies and standard pathways; outpatient prescriptions depend on step-down practices.
• Formulation mix: oral modern formulations generally support substitution away from suspension dosing complexity and drive adherence.
• Tendering and reimbursement: generic penetration in many markets compresses price; branded pricing persists where exclusivity or formulation protection remains.

Commercial takeaway Revenue tends to track:

• patient-risk incidence and guideline adherence;
• formulary persistence and switching cycles;
• genericization and procurement price compression.

---

Forecast: base-case projection logic for revenue and volume

What drives a realistic 3 to 5-year outlook?

A defensible forecast for posaconazole is built from three levers:

1. Epidemiology and risk cohort sizing
   • stable growth in treated oncology patients increases prophylaxis opportunity.
2. Share stability versus substitution
   • isavuconazole and echinocandins pressure selection in some sub-indications.
3. Pricing trend from competition
   • generic and tendering dynamics reduce net pricing over time.

Resulting base-case market shape

• Revenue growth tends to be modest and driven more by volume and guideline use than by premium price.
• Volume growth can outrun pricing declines in some regions if prophylaxis penetration expands.
• In mature markets, revenue often flattens because price compression outpaces incidence growth.

---

Market projection by scenario (directional, clinically anchored)

Because precise, audited market sizing for “posaconazole” varies by source methodology (scope: prophylaxis inclusion, formulation inclusion, and geography), the most actionable forecast framework is scenario-based directionality.

Scenario	Revenue trajectory	Primary cause
Base case	low-to-moderate growth	sustained prophylaxis volumes plus continued formulary use; pricing compression partially offset
Bull case	higher growth	stronger prophylaxis penetration and step-down outpatient adoption; slower switching due to clinician preference and tolerability outcomes
Bear case	flat to decline	accelerated substitution toward other triazoles/echinocandins; aggressive procurement and generic price drops

Business interpretation Under all scenarios, posaconazole remains a “core” IFD prophylaxis/therapy anchor. The upside comes from market penetration and care pathway optimization, while the downside comes from substitution and procurement economics.

---

Clinical trials update: what to watch next

Which trial signal types matter most for commercial impact?

For a mature active ingredient like posaconazole, the commercially meaningful signals are the ones that change prescribing behavior. Those typically include:

• Prophylaxis breakthrough infection reduction in defined high-risk subgroups.
• Safety comparability that supports broader outpatient step-down and longer-duration prophylaxis.
• Therapeutic drug monitoring (TDM) operational evidence that reduces clinician friction and improves real-world attainment.
• Formulation usability and adherence data that reduces IV burden and supports protocol-driven prophylaxis.

What to discount Signals that only replicate prior efficacy without changing dosing, patient selection, or operational feasibility usually do not move market share.

---

Key regulatory and guideline positioning

How guideline placement supports durability

Posaconazole maintains durable market position because it aligns with standard care in:

• high-risk antifungal prophylaxis for hematologic malignancies and transplant recipients;
• first-line or preferred options in certain IFD contexts where oral therapy is feasible.

Guideline adherence is a strong determinant of steady demand because hematology protocols standardize antifungal selection.

---

Key Takeaways

• Posaconazole demand is anchored to high-risk prophylaxis and established IFD treatment pathways rather than a rapidly expanding late-stage pipeline under the same INN umbrella.
• Commercial growth is driven primarily by patient-risk cohort sizing and prophylaxis penetration, not by major indication breakthroughs.
• The competitive threat is substitution within the antifungal class ecosystem (notably other oral triazoles and echinocandins) plus ongoing pricing pressure from procurement and genericization.
• The most commercially relevant trial signals are those that reduce breakthrough infections, expand usable populations, and improve operational feasibility (including TDM and step-down).

---

FAQs

1) Is posaconazole growth more dependent on prophylaxis or treatment?

Prophylaxis typically drives volume in hematology/transplant care pathways, while treatment drives the higher-acuity but lower-incidence share of spend. Net market outcomes usually track prophylaxis penetration and protocol adherence.

2) What endpoints matter most for market-changing evidence in posaconazole?

Breakthrough IFD rates, overall response in key IFD categories, survival in severe disease strata, and safety/tolerability signals that support broader prescribing.

3) What is the main competitive risk to posaconazole?

Substitution within the oral triazole class and shifts toward echinocandin-based strategies in overlapping settings, reinforced by formulary and procurement decisions.

4) How do pricing dynamics typically affect posaconazole revenue?

Generic and tender pricing compress net revenue over time; volume growth from prophylaxis uptake can offset but often cannot fully counter price declines in mature markets.

5) What trial signals should investors prioritize?

Studies that change operational prescribing behavior: improved adherence and exposure, reduced breakthrough infections in defined subgroups, and safety results supporting expanded prophylaxis durations or outpatient step-down.

---

References

[1] Pfizer Inc. Noxafil (posaconazole) prescribing information. (US label).
[2] EMA. Noxafil: Summary of Product Characteristics (posaconazole).
[3] IDSA (Infectious Diseases Society of America). Guidelines for the diagnosis and management of aspergillosis and candidiasis (latest versions available on IDSA).
[4] ECIL (European Conference on Infections in Leukaemia). Guidelines for antifungal prophylaxis in leukemia and stem cell transplant (latest available versions).
[5] NCCN. Supportive Care / Antimicrobial prophylaxis and treatment guidance (latest available version).