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Data updated: Mar 10, 2026

MYLOTARG

GEMTUZUMAB OZOGAMICIN CD33-directed Antibody Interactions
Oncology Approved 2017-09-01

Mylotarg is a CD33-directed antibody-drug conjugate used to treat CD33-positive acute myeloid leukemia (AML). It is indicated for newly-diagnosed patients, including adults and pediatric patients as young as one month old. Additionally, the drug is approved for adults and pediatric patients aged two years and older with relapsed or refractory CD33-positive AML. This therapeutic provides a targeted approach for both treatment-naive and previously treated AML populations.

Source: FDA Label • Pfizer • CD33-directed Immunoconjugate

How MYLOTARG Works

Mylotarg consists of a monoclonal antibody linked to a cytotoxic agent, N-acetyl gamma calicheamicin. The antibody portion targets and binds to the CD33 antigen expressed on the surface of tumor cells, which then triggers the internalization of the drug complex. Once inside the cell, the cytotoxic agent is released through the cleavage of its linker, leading to double-strand DNA breaks. This process results in cell cycle arrest and the programmed death of the leukemia cells.

Source: FDA Label
2
Indications
--
Phase 3 Trials
1
Priority Reviews
8
Years on Market

Details

Status
Prescription
First Approved
2017-09-01
Routes
SINGLE-DOSE
Dosage Forms
VIAL

Companies

Active Ingredient: GEMTUZUMAB OZOGAMICIN

MYLOTARG Approval History

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What MYLOTARG Treats

1 indications

MYLOTARG is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Acute Myeloid Leukemia
Source: FDA Label

MYLOTARG Boxed Warning

HEPATOTOXICITY Hepatotoxicity, including severe or fatal hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), has been reported in association with the use of MYLOTARG as a single agent, and as part of a combination chemotherapy regimen. Monitor frequently for signs and symptoms of VOD after treatment with MYLOTARG. ( 5.1 and 6.1 ) WARNING: HEPATOTOXICITY See full prescribing information for complete boxed warning. Hepatotoxicity, including severe or fatal h...

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Trial Timeline

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Understanding FDA Approval Types
Count Type What it means
- ORIG Original approval - drug first enters market
- SUPPL - Efficacy New indication (new disease/condition approved)
- SUPPL - Labeling Label text changes (warnings, dosing updates)
- SUPPL - Manufacturing Production changes (new facility)
- SUPPL - Chemistry Formulation changes (new dosage strength)

Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.

MYLOTARG FDA Label Details

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Indications & Usage

FDA Label (PDF)

MYLOTARG is a CD33-directed antibody and cytotoxic drug conjugate indicated for: • treatment of newly-diagnosed CD33-positive acute myeloid leukemia (AML) in adults and pediatric patients 1 month and older . • treatment of relapsed or refractory CD33-positive AML in adults and pediatric patients 2 years and older . 1.1 Newly-Diagnosed CD33-positive Acute Myeloid Leukemia (AML) MYLOTARG is indicated for the treatment of newly-diagnosed CD33-positive acute myeloid leukemia in adults and pediatric patients 1 month and older. 1.2 Relapsed or Refractory CD33-positive AML MYLOTARG is indicated for t...

⚠️ BOXED WARNING

WARNING: HEPATOTOXICITY Hepatotoxicity, including severe or fatal hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), has been reported in association with the use of MYLOTARG as a single agent, and as part of a combination chemotherapy regimen. Monitor frequen...

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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.