CLINICAL TRIALS PROFILE FOR GEMTUZUMAB OZOGAMICIN

All Clinical Trials for Gemtuzumab Ozogamicin

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00003131 ↗	CMA-676 in Treating Patients With Acute Myeloid Leukemia in First Relapse	Completed	Pfizer	Phase 2	1997-02-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of CMA-676 in treating patients with acute myeloid leukemia in first relapse.
NCT00003673 ↗	CMA-676 in Treating Older Patients With Acute Myeloid Leukemia in First Relapse	Completed	Pfizer	Phase 2	1998-03-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase II trial to study the effectiveness of CMA-676 in treating older patients who have acute myeloid leukemia that has recurred for the first time following at least 3 months of complete remission.
NCT00005962 ↗	Comparison of Three Treatment Regimens in Treating Patients With Relapsed or Refractory Acute Myelogenous Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	2000-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Monoclonal antibodies such as gemtuzumab ozogamicin can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Combining more than one drug or combining monoclonal antibody with chemotherapy may kill more cancer cells. It is not yet known which treatment regimen is more effective for acute myelogenous leukemia. PURPOSE: Randomized phase II trial to compare the effectiveness of three treatment regimens in treating patients who have relapsed or refractory acute myelogenous leukemia.
NCT00005962 ↗	Comparison of Three Treatment Regimens in Treating Patients With Relapsed or Refractory Acute Myelogenous Leukemia	Completed	Eastern Cooperative Oncology Group	Phase 2	2000-07-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Monoclonal antibodies such as gemtuzumab ozogamicin can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Combining more than one drug or combining monoclonal antibody with chemotherapy may kill more cancer cells. It is not yet known which treatment regimen is more effective for acute myelogenous leukemia. PURPOSE: Randomized phase II trial to compare the effectiveness of three treatment regimens in treating patients who have relapsed or refractory acute myelogenous leukemia.
NCT00006122 ↗	Gemtuzumab Ozogamicin With or Without Chemotherapy in Treating Older Patients With Acute Myeloid Leukemia	Completed	European Organisation for Research and Treatment of Cancer - EORTC	Phase 2	2000-06-01	RATIONALE: Monoclonal antibodies such as gemtuzumab ozogamicin can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Drugs used in chemotherapy uses different ways to stop cancer cells from dividing so they stop growing or die. Combining gemtuzumab ozogamicin with chemotherapy may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of gemtuzumab ozogamicin with or without chemotherapy in treating older patients who have acute myeloid leukemia.
NCT00006265 ↗	Gemtuzumab Ozogamicin and High-Dose Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	2001-03-01	RATIONALE: Monoclonal antibodies, such as gemtuzumab ozogamicin, can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Drugs used in chemotherapy, such as cytarabine, use different ways to stop cancer cells from dividing so they stop growing or die. Combining gemtuzumab ozogamicin with cytarabine may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining gemtuzumab ozogamicin with high-dose cytarabine in treating patients who have relapsed or refractory acute myeloid leukemia.
NCT00006265 ↗	Gemtuzumab Ozogamicin and High-Dose Cytarabine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia	Completed	Alliance for Clinical Trials in Oncology	Phase 2	2001-03-01	RATIONALE: Monoclonal antibodies, such as gemtuzumab ozogamicin, can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. Drugs used in chemotherapy, such as cytarabine, use different ways to stop cancer cells from dividing so they stop growing or die. Combining gemtuzumab ozogamicin with cytarabine may kill more cancer cells. PURPOSE: Phase II trial to study the effectiveness of combining gemtuzumab ozogamicin with high-dose cytarabine in treating patients who have relapsed or refractory acute myeloid leukemia.

Clinical Trial Conditions for Gemtuzumab Ozogamicin

Condition Name

Condition Name for Gemtuzumab Ozogamicin
Intervention	Trials
Acute Myeloid Leukemia	38
Leukemia	24
Myelodysplastic Syndrome	11
Acute Myelogenous Leukemia	9

Condition MeSH

Condition MeSH for Gemtuzumab Ozogamicin
Intervention	Trials
Leukemia	86
Leukemia, Myeloid, Acute	86
Leukemia, Myeloid	80
Myelodysplastic Syndromes	22

Clinical Trial Locations for Gemtuzumab Ozogamicin

Trials by Country

Trials by Country for Gemtuzumab Ozogamicin
Location	Trials
United States	500
Canada	33
Germany	15
United Kingdom	11
Australia	9

Trials by US State

Trials by US State for Gemtuzumab Ozogamicin
Location	Trials
Texas	26
New York	23
California	22
Washington	19
Michigan	18

Clinical Trial Progress for Gemtuzumab Ozogamicin

Clinical Trial Phase

Clinical Trial Phase for Gemtuzumab Ozogamicin
Clinical Trial Phase	Trials
PHASE3	1
PHASE2	2
PHASE1	2

Clinical Trial Status

Clinical Trial Status for Gemtuzumab Ozogamicin
Clinical Trial Phase	Trials
Completed	46
Recruiting	29
Terminated	6

Clinical Trial Sponsors for Gemtuzumab Ozogamicin

Sponsor Name

Sponsor Name for Gemtuzumab Ozogamicin
Sponsor	Trials
National Cancer Institute (NCI)	32
Pfizer	16
M.D. Anderson Cancer Center	12

Sponsor Type

Sponsor Type for Gemtuzumab Ozogamicin
Sponsor	Trials
Other	112
Industry	39
NIH	32

Clinical Trials Update, Market Analysis, and Projection for Gemtuzumab Ozogamicin

Last updated: October 30, 2025

Introduction

Gemtuzumab Ozogamicin (brand name Mylotarg) is an antibody-drug conjugate (ADC) approved for the treatment of acute myeloid leukemia (AML). Developed by Pfizer, it was initially approved in 2017, subsequently withdrawn in 2010 due to safety concerns, and re-approved in 2017 after clinical modifications. Its targeted mechanism involves an anti-CD33 antibody linked to a potent cytotoxic agent, calicheamicin, enabling selective killing of AML cells expressing CD33 antigen.

This article synthesizes recent clinical trial updates, provides a comprehensive market analysis, and forecasts future growth trajectories for Gemtuzumab Ozogamicin within the evolving oncology treatment landscape.

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Clinical Trial Landscape

Recent Clinical Trials and Updates

In recent years, the scope of Gemtuzumab Ozogamicin's clinical evaluation has expanded beyond its initial approval for newly diagnosed and relapsed AML. Key developments include:

• Combination Therapies: Multiple phase 1 and 2 trials are investigating Gemtuzumab Ozogamicin in combination with hypomethylating agents (e.g., azacitidine, decitabine). These combinations aim to improve remission rates in elderly and unfit patients with AML. A notable trial (NCT03459593) demonstrated encouraging response rates when combined with azacitidine in high-risk AML cohorts.

• Maintenance Therapy: Ongoing phase 3 trials are assessing its potential as a maintenance therapy post-induction chemotherapy. These studies aim to evaluate long-term survival benefits and minimal residual disease (MRD) clearance.

• Minimal Residual Disease (MRD) Targeting: Recent trials focus on using Gemtuzumab Ozogamicin in MRD-positive patients, seeking to prevent relapse and improve overall survival. Data from early-phase studies suggest that targeting MRD with ADCs like Gemtuzumab Ozogamicin holds promise.

• New Formulations & Dosing Regimens: Research efforts are exploring optimized dosing schedules, including fractionated doses, to balance efficacy with toxicity, especially hepatotoxicity (e.g., veno-occlusive disease).

Key Clinical Trial Outcomes Summary:

Trial Focus	Phase	Outcomes	Significance
Combination with azacitidine	Phase 1/2	Higher CR rates	Supports combination regimens
Maintenance therapy post-remission	Phase 3	Pending final results	Expected to inform guidelines
MRD-targeted therapy	Phase 2	MRD clearance	Potential for relapse prevention

Regulatory and Safety Considerations

The re-approval process emphasized refined dosing to mitigate adverse events such as hepatotoxicity and myelosuppression. Post-marketing surveillance continues to monitor long-term safety, especially in frail populations.

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Market Analysis

Market Landscape and Competitive Position

Gemtuzumab Ozogamicin occupies a unique niche as a targeted therapy for CD33-positive AML. The global AML therapeutics market was valued at approximately USD 725 million in 2022, with high-growth potential driven by the emergence of targeted agents and personalized medicine approaches [1].

Key competitors include:

• CPX-351 (Vyxeos): A liposomal formulation of cytarabine and daunorubicin, approved for therapy-related AML and secondary AML. It competes by offering a different mechanistic approach but targets the same patient subset.

• Venetoclax-based regimens: Particularly in elderly AML, combining BCL-2 inhibitors with hypomethylating agents is gaining traction.

• Other CD33-targeted agents: Although limited, experimental agents and biosimilars are under development.

Despite these competitors, Gemtuzumab Ozogamicin’s specificity for CD33-positive AML provides a targeted niche, especially in patients unfit for intensive chemotherapy.

Market Drivers

• Rising incidence of AML globally, particularly in aging populations.

• Increasing FDA approvals of combination regimens involving Gemtuzumab Ozogamicin.

• Growing emphasis on targeted therapies and precision medicine in oncology.

• Expansion of clinical indications, including use as maintenance or in MRD-positive cases.

Market Challenges

• Toxicity concerns, primarily hepatotoxicity and myelosuppression.

• Strict patient selection criteria due to safety profile.

• Preference for combination regimens that may dilute direct sales of standalone Gemtuzumab Ozogamicin.

• Regulatory hurdles in emerging markets.

Regional Market Insights

North America dominates due to high clinical adoption rates, robust healthcare infrastructure, and ongoing clinical trials. Europe follows, with increased adoption in standard-of-care protocols. The Asia-Pacific region presents opportunities due to rising AML incidence and expanding healthcare investments, though regulatory and reimbursement hurdles persist.

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Market Projection

Forecast Overview

The global market for Gemtuzumab Ozogamicin is projected to grow at a Compound Annual Growth Rate (CAGR) of approximately 8-10% over the next five years, reaching an estimated USD 1.2 billion by 2028.

Key Factors Influencing Growth

• Clinical Expansion: Ongoing trials demonstrating efficacy in MRD, maintenance therapy, and combination regimens are likely to broaden approved indications and increase usage.

• Regulatory Approvals: Anticipated approvals in additional countries, especially emerging markets, will widen patient access.

• Technological Advancements: Improvements in ADC design, dosing optimization, and safety profile may enhance adoption rates.

• Patient Demographics: Aging populations globally are leading to increased AML diagnoses, directly influencing market growth.

Potential Market Risks

• Emergence of superior or safer competitors.

• Evolving regulatory standards that may restrict usage or require additional trials.

• Economic factors impacting healthcare expenditure and reimbursement policies.

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Key Takeaways

• Clinical trials continue to expand Gemtuzumab Ozogamicin’s therapeutic scope, with promising results in combination therapy, MRD targeting, and maintenance settings.

• Market dynamics favor growth driven by increasing AML incidence, favorable clinical outcomes, and a growing preference for targeted therapies.

• Safety management remains crucial, with ongoing refinements in dosing regimens to mitigate adverse effects.

• Future projections indicate steady growth, with the market expected to reach approximately USD 1.2 billion by 2028, supported by regulatory approvals and clinical validation.

• Strategic positioning for stakeholders involves ongoing clinical development, safety profiling, and expansion into emerging markets.

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FAQs

1. What are the main indications for Gemtuzumab Ozogamicin?
   It is primarily indicated for CD33-positive acute myeloid leukemia, including newly diagnosed and relapsed cases, with ongoing research into maintenance and combination therapies.

2. How does Gemtuzumab Ozogamicin compare with other AML treatments?
   It offers targeted therapy with a unique mechanism as an ADC, providing an option for patients unfit for intensive chemotherapy, and showing improved remission rates when combined with hypomethylating agents.

3. What are the safety concerns associated with Gemtuzumab Ozogamicin?
   Key safety issues include hepatotoxicity, particularly veno-occlusive disease, hematologic toxicities, and infusion-related reactions. Dose optimization and patient monitoring are critical.

4. Are there ongoing clinical trials that could expand Gemtuzumab Ozogamicin's approved uses?
   Yes, several phase 2 and 3 trials are exploring its role in MRD management, maintenance therapy, and in combination with novel agents, which could lead to expanded indications.

5. What is the outlook for Gemtuzumab Ozogamicin’s market share?
   With continued clinical validation and regulatory support, Gemtuzumab Ozogamicin is poised for steady market growth, securing its position within targeted AML therapies.

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References

[1] Grand View Research. (2022). AML Therapeutics Market Size, Share & Trends Analysis.
[2] Pfizer Inc. (2017). FDA approves new indication for Mylotarg — re-approval report.
[3] ClinicalTrials.gov. Ongoing trials involving Gemtuzumab Ozogamicin.
[4] National Cancer Institute. (2022). AML Overview.