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
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.
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.
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.
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.
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.

Clinical Trial Conditions for AMBISOME

Condition Name

Condition Name for AMBISOME
Intervention	Trials
Visceral Leishmaniasis	11
Leishmaniasis	3
Invasive Aspergillosis	3
Aspergillosis	3

Condition MeSH

Condition MeSH for AMBISOME
Intervention	Trials
Leishmaniasis	22
Leishmaniasis, Visceral	18
Mycoses	11
Aspergillosis	10

Clinical Trial Locations for AMBISOME

Trials by Country

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

Trials by US State

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−

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

Clinical Trial Progress for AMBISOME

Clinical Trial Phase

Clinical Trial Phase for AMBISOME
Clinical Trial Phase	Trials
Phase 4	14
Phase 3	17
Phase 2/Phase 3	2

Clinical Trial Status

Clinical Trial Status for AMBISOME
Clinical Trial Phase	Trials
Completed	43
Terminated	7
Recruiting	3

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	66
Industry	33
U.S. Fed	6

Clinical Trials, Market Analysis, and Projections for AMBISOME

Introduction

AMBISOME, a liposomal formulation of amphotericin B, has been a cornerstone in the treatment of severe fungal infections, particularly in immunocompromised patients. This article delves into the recent clinical trials, market analysis, and future projections for this critical antifungal medication.

Clinical Trials: The AMBITION-cm Regimen

Background and Trial Design

The AMBITION-cm trial, a phase III randomized clinical trial, has been pivotal in shaping the current treatment guidelines for HIV-associated cryptococcal meningitis. This trial compared the efficacy and safety of a single 10 mg/kg dose of liposomal amphotericin B (AMBISOME) combined with 14 days of flucytosine and fluconazole against the standard of care, which involved amphotericin B deoxycholate at 1 mg/kg/day plus flucytosine for 7 days, followed by fluconazole for 7 days[1][3].

Key Findings

• Efficacy: The AMBITION-cm trial demonstrated that the single-dose AMBISOME regimen was non-inferior to the standard of care in terms of 10-week survival. The trial showed a 10-week survival probability of 71.7% in the AMBISOME group, compared to 68.6% in the observational cohort[1].
• Safety: The AMBISOME regimen was associated with fewer adverse events, particularly infusion-related reactions and nephrotoxicity, compared to conventional amphotericin B[1][4].
• Cost-Effectiveness: The regimen was found to be cost-effective, with incremental cost-effectiveness ratios ranging from $71 to $121 per life-year gained, making it a viable option for low- and middle-income settings[3].

Real-World Implementation

Outcomes in Routine Care

Following the WHO's 2022 guidelines recommending the AMBITION-cm regimen, real-world implementation in Uganda showed promising results. The 10-week survival rate in the observational cohort was 68.6%, which was comparable to the clinical trial outcomes. This indicates that the regimen is effective and feasible in routine care settings[1].

Market Analysis

Market Size and Growth

The AMBISOME market has been growing at a moderate pace, driven by the increasing incidence of fungal infections, particularly in immunocompromised patients such as those with HIV/AIDS, cancer, and organ transplant recipients. The market is expected to grow significantly from 2024 to 2031, fueled by rising healthcare expenditures and improving healthcare infrastructure in emerging markets[2].

Key Drivers

• Increasing Incidence of Fungal Infections: The growing population of immunocompromised individuals and the rising awareness among healthcare professionals are key drivers of the AMBISOME market[2].
• Favorable Regulatory Environment: Expanding healthcare infrastructure and favorable regulatory environments in emerging markets are also driving the market growth[2].

Market Segmentation

The AMBISOME market is segmented by product type, application, end-user, and geography. Hospitals form the largest end-user segment, followed by specialty clinics and homecare. Geographically, the market is divided into North America, Europe, Asia-Pacific, Latin America, and the Middle East and Africa, each with unique healthcare dynamics and market opportunities[2].

Competitive Landscape

Major Players

The AMBISOME market is dominated by several key players, including Gilead Sciences, Cipla, Bristol-Myers Squibb Company, Matinas Biopharma Holdings, and others. These companies are involved in the development, marketing, and distribution of AMBISOME and other antifungal medications[2].

Challenges and Opportunities

• Generic Competition: The market faces significant competition from generic formulations, which can limit the market share of branded drugs like AMBISOME. However, the unique benefits and safety profile of AMBISOME help it maintain a strong market position[2].
• Expanding Healthcare Access: The ongoing expansion of healthcare infrastructure in emerging markets presents opportunities for increased penetration and use of AMBISOME[2].

Cost-Effectiveness and Economic Implications

Economic Analysis

The cost-effectiveness analysis of the AMBITION-cm regimen has shown that it is highly cost-effective, especially in low- and middle-income settings. The incremental cost-effectiveness ratios indicate that the regimen can be cost-saving, particularly when excluding protocol-driven costs[3].

Advocacy and Access

The economic evidence supports advocacy efforts to ensure that essential medicines like AMBISOME and flucytosine are made available at agreed access prices, facilitating widespread implementation of the AMBITION-cm regimen[3].

Future Projections

Market Growth

The AMBISOME market is projected to continue growing, driven by increasing demand for effective antifungal treatments, expanding healthcare infrastructure, and favorable regulatory environments. The Asia-Pacific region is expected to show rapid market growth due to rising healthcare expenditures and a growing population at risk of fungal infections[2].

Clinical and Economic Impact

The widespread adoption of the AMBITION-cm regimen is expected to improve treatment outcomes for patients with HIV-associated cryptococcal meningitis, reducing mortality rates and improving the quality of care. The cost-effectiveness of the regimen will be crucial in sustaining its use in resource-limited settings[1][3].

Key Takeaways

• Clinical Efficacy: The AMBITION-cm regimen has demonstrated non-inferiority and improved safety compared to the standard of care.
• Real-World Implementation: The regimen has shown promising results in routine care settings.
• Market Growth: The AMBISOME market is expected to grow significantly, driven by increasing demand and expanding healthcare infrastructure.
• Cost-Effectiveness: The regimen is highly cost-effective, particularly in low- and middle-income settings.
• Competitive Landscape: The market faces competition from generics but maintains a strong position due to its unique benefits.

FAQs

What is the AMBITION-cm regimen?

The AMBITION-cm regimen involves a single 10 mg/kg dose of liposomal amphotericin B (AMBISOME) combined with 14 days of flucytosine and fluconazole for the treatment of HIV-associated cryptococcal meningitis.

How effective is the AMBITION-cm regimen?

The regimen has been shown to be non-inferior to the standard of care in terms of 10-week survival and has a better safety profile, with fewer adverse events.

What are the key drivers of the AMBISOME market?

The increasing incidence of fungal infections, favorable regulatory environments, and expanding healthcare infrastructure in emerging markets are key drivers.

Who are the major players in the AMBISOME market?

Major players include Gilead Sciences, Cipla, Bristol-Myers Squibb Company, and others.

Is the AMBITION-cm regimen cost-effective?

Yes, the regimen has been found to be highly cost-effective, particularly in low- and middle-income settings, with incremental cost-effectiveness ratios indicating potential cost savings.

Sources

1. Implementation of Single High-dose Liposomal Amphotericin B ... - Academic.oup.com
2. Ambisome Market Size, Scope, Growth, Trends and Forecast - Verifiedmarketresearch.com
3. Cost-effectiveness of single, high-dose, liposomal amphotericin ... - The Lancet
4. AmBisome® Phase III Study For Cryptococcal Meningitis - Gilead.com