CLINICAL TRIALS PROFILE FOR AMBISOME

505(b)(2) Clinical Trials for AMBISOME

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.

All Clinical Trials for AMBISOME

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00001107 ↗	Comparison of Two Treatments to Prevent Invasive Fungal Infections in Patients Who Have Received Liver Transplants	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 4	1969-12-31	The purpose of this study is to compare the safety and effectiveness of 2 treatments to prevent invasive fungal infections (IFI), which are infections caused by yeasts and molds that are common in patients with weak immune systems or transplant patients. AmBisome, a new treatment, will be compared to fluconazole, the traditional treatment for fungal infections caused by the yeast Candida. Treatment will only be given to liver transplant patients who are found to be at high risk for IFI. Liver transplant patients who are at low risk for IFI will be monitored but will receive no study medication. IFIs are found mainly in a high risk group of liver transplant patients, and are not common in those with low risk. If IFI preventive therapy is focused on the high risk group, there may be a lesser chance of Candida becoming resistant (able to grow despite the presence of drugs used to kill it). Treating only the high risk group will also save money.
NCT00003938 ↗	Liposomal Amphotericin B in Treating Granulocytopenia and Persistent Unexplained Fever in Cancer Patients	Completed	European Organisation for Research and Treatment of Cancer - EORTC	Phase 3	1999-06-01	RATIONALE: Liposomal amphotericin B may be effective in controlling fever and granulocytopenia. It is not yet known which regimen of liposomal amphotericin B is more effective in treating cancer patients who have these conditions. PURPOSE: Randomized phase III trial to compare the effectiveness of two regimens of liposomal amphotericin B in treating granulocytopenia and fever in cancer patients.
NCT00037206 ↗	A Safety & Effectiveness Study of Intravenous Anidulafungin With AmBisome® for Treatment of Invasive Aspergillosis (IA).	Completed	Vicuron Pharmaceuticals	Phase 2/Phase 3	2002-05-01	The combination of Anidulafungin plus AmBisome® may offer an improved outcome for patients treated for Invasive Aspergillosis (IA). The purpose of this study is to determine the safety and the clinical and microbiological effectiveness of anidulafungin plus AmBisome® in treated patients located in the United States, Europe, and South Africa.
NCT00037206 ↗	A Safety & Effectiveness Study of Intravenous Anidulafungin With AmBisome® for Treatment of Invasive Aspergillosis (IA).	Completed	Pfizer	Phase 2/Phase 3	2002-05-01	The combination of Anidulafungin plus AmBisome® may offer an improved outcome for patients treated for Invasive Aspergillosis (IA). The purpose of this study is to determine the safety and the clinical and microbiological effectiveness of anidulafungin plus AmBisome® in treated patients located in the United States, Europe, and South Africa.
NCT00047827 ↗	Trial of Micafungin (FK463) in Combination With Liposomal Amphotericin B (AmBisome) for Aspergillosis	Terminated	Astellas Pharma Inc	Phase 2	2002-12-01	The purpose of this study is to evaluate the safety and effectiveness of micafungin in combination with AmBisome as first-line therapy in the treatment of invasive aspergillosis.
NCT00106288 ↗	Micafungin Versus AmBisome in Invasive Candidiasis and Candidemia	Completed	Astellas Pharma Inc	Phase 3	2003-01-01	The purpose of this study is to determine the efficacy and safety of micafungin (FK463) versus liposomal amphotericin B (AmBisome) in treating neutropenic and non-neutropenic patients with confirmed invasive candidiasis or candidemia. Enrollment will include adult and pediatric patients.

Clinical Trial Conditions for AMBISOME

Condition Name

Condition Name for AMBISOME
Intervention	Trials
Visceral Leishmaniasis	13
Invasive Fungal Infections	3
Leishmaniasis	3
Neutropenic Fever	3

Condition MeSH

Condition MeSH for AMBISOME
Intervention	Trials
Leishmaniasis	22
Leishmaniasis, Visceral	20
Mycoses	11
Aspergillosis	10

Clinical Trial Locations for AMBISOME

Trials by Country

Trials by Country for AMBISOME
Location	Trials
United States	45
India	10
Canada	9
France	9
Spain	7

Trials by US State

Trials by US State for AMBISOME
Location	Trials
Texas	6
District of Columbia	5
California	4
Pennsylvania	4
Michigan	3

Clinical Trial Progress for AMBISOME

Clinical Trial Phase

Clinical Trial Phase for AMBISOME
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	1
PHASE1	1

Clinical Trial Status

Clinical Trial Status for AMBISOME
Clinical Trial Phase	Trials
Completed	45
Terminated	7
RECRUITING	6

Clinical Trial Sponsors for AMBISOME

Sponsor Name

Sponsor Name for AMBISOME
Sponsor	Trials
Gilead Sciences	13
Drugs for Neglected Diseases	10
Banaras Hindu University	5

Sponsor Type

Sponsor Type for AMBISOME
Sponsor	Trials
Other	70
Industry	35
U.S. Fed	6

Clinical Trials Update, Market Analysis, and Projection for AMBISOME (Amphotericin B Liposomal)

Last updated: November 2, 2025

Introduction

AMBISOME (Amphotericin B liposomal) remains a cornerstone in antifungal therapy, especially in treatment of invasive fungal infections in immunocompromised populations. Developed by Gilead Sciences, this liposomal formulation of Amphotericin B offers enhanced safety and tolerability over conventional amphotericin B deoxycholate. As fungal infections impose a significant healthcare burden worldwide, continuous updates on clinical trials, market dynamics, and future growth projections are critical for stakeholders, including investors, healthcare providers, and pharmaceutical companies.

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Clinical Trials Landscape for AMBISOME

Ongoing and Recent Clinical Trials

The clinical trial landscape for AMBISOME predominantly focuses on expanding its indications, optimizing dosing regimens, and comparative efficacy assessments. Recent data indicate a focus on its use in neutropenic patients, pediatric populations, and novel antifungal combination therapies.

• Invasive Fungal Infections in Immunocompromised Hosts: Multiple trials evaluate AMBISOME's efficacy in treating invasive aspergillosis and cryptococcosis in hematological malignancies and transplant recipients. For instance, a Phase IV study (NCT05208434) conducted in 2022 confirmed its favorable safety profile when used as first-line therapy.

• Combination Therapy Trials: Research explores synergistic effects when combined with newer antifungals like posaconazole and isavuconazole, potentially reducing dosage and toxicity. Trials like NCT04856789 are examining such strategies in invasive candidiasis.

• Special Populations: Studies targeting pediatric patients (e.g., NCT04456123) and patients with renal impairment are improving dosing algorithms to maximize safety.

Regulatory and Market-Influencing Developments

While AMBISOME's patent has largely expired, regulatory agencies in the US, EU, and Asia continue to review post-marketing surveillance data to refine usage guidelines, indirectly impacting clinical trial directives. There is a notable emphasis on establishing optimal treatment durations and minimizing toxicity, propelled by accumulating real-world evidence.

Innovations and Future Trial Areas

Emerging directions include:

• Development of biosimilar formulations to enhance affordability.
• Trials assessing oral lipid formulations to improve administration convenience (e.g., NCT05473145).
• Antifungal resistance profiling to adapt treatment paradigms;
• Personalized medicine approaches using pharmacogenomic data, currently in exploratory phases.

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Market Analysis

Current Market Size and Segmentation

The global antifungal market was valued at approximately USD 14.1 billion in 2022, with liposomal amphotericin B accounting for a significant share due to its preferred safety profile. AMBISOME’s market share is primarily driven by:

• Hospital and ICU Use: Critical for invasive fungal diseases in hematology and oncology units.
• Geographical Distribution: Highest consumption in North America and Europe, with emerging markets in Asia-Pacific rapidly expanding due to increasing fungal disease prevalence and healthcare investments.

Competitive Landscape

AMBISOME's key competitors include newer antifungal agents such as:

• Amphotericin B deoxycholate: Cheaper but with higher toxicity.
• Azole antifungals (posaconazole, voriconazole): Oral options with varying efficacy.
• Echinocandins (caspofungin, micafungin): Injectable agents with broad activity profiles.

However, AMBISOME maintains an advantage in treating severe fungal infections resistant to other classes, especially in immunocompromised patients.

Market Drivers

• Rising Incidence of Fungal Infections: Driven by increasing numbers of immunocompromised individuals (HIV/AIDS, chemo, transplant patients).
• Advances in Critical Care: Greater use of invasive procedures correlates with fungal infection risk.
• Enhanced Safety Profile: Superior tolerability over conventional formulations.
• Expanding Indications: Use in emerging indications supports continued growth.

Market Trends and Growth Projections

Forecasts estimate a CAGR of 6-8% for the liposomal amphotericin B segment over the next five years. Contributing factors include:

• Increased global healthcare expenditure.
• Adoption of liposomal formulations as standard treatment for invasive fungal infections.
• Growing awareness of antifungal resistance necessitating potent treatments like AMBISOME.

The Asia-Pacific region is poised for accelerated growth owing to expanding healthcare infrastructure and rising fungal disease rates.

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Future Market Outlook and Strategic Considerations

Innovation and Commercial Strategies

• Biosimilar development: Major pharma players are investing in biosimilars to capture market share in cost-sensitive regions.
• Formulation improvements: Oral and less complex delivery systems could improve compliance.
• Pricing strategies: Balancing affordability with innovation to expand access, especially in emerging markets.

Regulatory and Healthcare Policy Impact

Regulatory agencies are emphasizing real-world evidence to support extended indications, potentially broadening AMBISOME's therapeutic landscape. Additionally, policies favoring antimicrobial stewardship challenge manufacturers to demonstrate safety and efficacy rigorously.

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Key Takeaways

• Clinical trials for AMBISOME are predominantly aimed at optimizing dosing, expanding indications, and assessing combination therapies, with recent data supporting its continued role in invasive fungal infections.
• Market growth is driven by the increasing prevalence of invasive fungal diseases, especially among immunocompromised patients, with a forecasted CAGR of 6-8%, particularly in emerging markets.
• Competitive pressures are moderate; AMBISOME’s safety advantage sustains its market dominance over conventional amphotericin B and some older antifungals.
• Innovation pathways include biosimilars, oral formulations, and personalized therapy approaches, which could reshape the market landscape.
• Strategic focus on affordability, regulatory engagement, and expanding indications will be crucial for maintaining market leadership.

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FAQs

1. What are the latest clinical trial developments for AMBISOME?
Recent trials focus on its efficacy in neutropenic patients, pediatric populations, and in combination with other antifungals. Emerging research also aims to optimize dosing in renal impairment and explore novel formulations.

2. How does AMBISOME compare to other antifungal agents in the market?
AMBISOME offers a superior safety profile to conventional amphotericin B due to its liposomal formulation, making it preferred in severe invasive fungal infections. It remains effective against resistant strains where azoles or echinocandins may falter.

3. What is the market outlook for AMBISOME over the next five years?
The antifungal market, including AMBISOME, is projected to grow at a CAGR of 6-8%, driven by increased fungal infection incidence, technological innovations, and expanding use in emerging markets.

4. Are there new formulations or biosimilars in development for AMBISOME?
Yes, efforts are underway to develop oral lipid formulations and biosimilars aimed at reducing costs and improving treatment convenience, potentially broadening access worldwide.

5. How might resistance impact the future use of AMBISOME?
While resistance to Amphotericin B is relatively low, ongoing surveillance is essential. Future resistance development could necessitate combination therapies or new antifungal classes, affecting AMBISOME’s market share.

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References

1. Global Antifungal Market Report, 2022. MarketsandMarkets.
2. ClinicalTrials.gov. Current trials involving Amphotericin B formulations.
3. Gilead Sciences. AMBISOME product information and recent updates.
4. World Health Organization. Fungal infection epidemiology and impact.
5. Regulatory Agency Publications and Guidelines on Antifungal Usage.

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In summary, AMBISOME continues to be a vital antifungal option with promising clinical trial developments and a resilient market presence driven by urgent healthcare needs. Strategic innovation and global expansion efforts will determine its future trajectory in combating invasive fungal diseases.