MYLOTARG (gemtuzumab ozogamicin)
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.
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.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2017-09-01
- Patent Cliff
- 2024
- Routes
- SINGLE-DOSE
- Dosage Forms
- VIAL
MYLOTARG Approval History
What MYLOTARG Treats
1 indicationsMYLOTARG 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
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...
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 hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), has been reported in association with the use of MYLOTARG. ( 5.1 , 6.1 )
MYLOTARG Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in MYLOTARG's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications MYLOTARG treats. First-in-class if their pivotal trials read out positive.
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Clinical Trial Registry
38 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05564390 | NCI-2022-07006 NCI-2022-07006, MYELOMATCH | Ph 2 | recruiting | MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) |
| NCT04293562 | AAML1831 NCI-2020-00546, AAML1831 | Ph 3 | recruiting | A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations |
| NCT01409161 | 2010-0981 NCI-2011-02767, 2010-0981 | Ph 2 | recruiting | Tretinoin and Arsenic Trioxide With or Without Gemtuzumab Ozogamicin in Treating Patients With Previously Untreated Acute Promyelocytic Leukemia |
| NCT00658814 results posted | NCI-2009-00790 NCI-2009-00790, SWOG-S0703 | Ph 2 | active not recruiting | Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia |
| NCT05183035 | ITCC-101/APAL2020D 2021-003212-11, 2023-510160-12-00 | Ph 3 | recruiting | Venetoclax in Children With Relapsed Acute Myeloid Leukemia (AML) |
| NCT06917911 | NCI-2025-02426 NCI-2025-02426, MM1YA-A04 | Ph 2 | not yet recruiting | Testing the Addition of Venetoclax or Gemtuzumab Ozogamicin (GO) to Usual Treatment Regimen (Cytarabine and Daunorubicin, "7+3") for Core Binding Factor Acute Myeloid Leukemia (CBF-AML) to Improve Response (A MyeloMATCH Treatment Trial) |
| NCT05955261 | AML23 NCI-2023-04138 | Ph 2 | suspended | A Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia |
| NCT05558124 | MCC-21450 | Ph 1 | recruiting | CPX-351 in Combination With Gemtuzumab Ozogamicin in Newly Diagnosed AML |
| NCT04915612 | 2020-0484 NCI-2020-13915, 2020-0484 | Ph 1 | completed | Liposomal Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin for the Treatment of Relapsed Refractory Pediatric Patients With Acute Myeloid Leukemia |
| NCT03672539 | 2018-0235 NCI-2018-01812, 2018-0235 | Ph 2 | active not recruiting | Liposome-encapsulated Daunorubicin-Cytarabine and Gemtuzumab Ozogamicin in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia (AML) or High Risk Myelodysplastic Syndrome |
| NCT03737955 | RG1018001 NCI-2018-01613, 9966 | Ph 2 | recruiting | Fractionated Gemtuzumab Ozogamicin in Treating Measurable Residual Disease in Patients With Acute Myeloid Leukemia |
| NCT03589729 | 2017-0937 NCI-2018-01108, 2017-0937 | Ph 2 | recruiting | Dexrazoxane Hydrochloride in Preventing Heart-Related Side Effects of Chemotherapy in Participants With Blood Cancers |
| NCT03900949 | OSU-21190 NCI-2019-01726 | Ph 1 | completed | Gentuzumab Ozogamicin and Midostaurin Combination With Standard Cytarabine and Danunorubi Midostaurin as a Novel Approach to Treating Patients With Newly Diagnosed FLT-3 Mutated Acute Myeloid Leukemia |
| NCT04849910 | VBP101 | Ph 1, Ph 2 | terminated | Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML or MDS |
| NCT04070768 results posted | BTCRC-AML17-113 | Ph 1 | completed | Study of the Safety and Efficacy of Gemtuzumab Ozogamicin (GO) and Venetoclax in Patients With Relapsed or Refractory CD33+ Acute Myeloid Leukemia:Big Ten Cancer Research Consortium BTCRC-AML17-113 |
| NCT05662904 GALAXY33 | 2022-002 | Ph 1 | not yet recruiting | Genetic Ablation of CD33 in HSC to Broaden the Therapeutic Index of CD33-directed Immunotherapy in Patients with AML |
| NCT05599360 | 0463-20 SZMC | Ph 2 | active not recruiting | Vyxeos for Induction of Low- or Intermediate-risk. |
| NCT04168502 | AML1819 | Ph 3 | recruiting | Gemtuzumab Chemotherapy MRD Levels; Adult Untreated, de Novo, Fav Interm Risk AML |
| NCT04207190 | I 435819 NCI-2019-07826, I 435819 | Ph 1 | completed | Talazoparib and Gemtuzumab Ozogamicin for the Treatment of CD33 Positive Relapsed or Refractory Acute Myeloid Leukemia |
| NCT04793919 | ICC APL STUDY 02 2017-002383-40 | Ph 2 | recruiting | Treatment Study for Children and Adolescents With Acute Promyelocytic Leukemia |
| NCT03904251 | 18-001419 NCI-2019-00701 | Ph 1 | terminated | CPX-351 and Gemtuzumab Ozogamicin in Treating Patients With Relapsed Acute Myeloid Leukemia |
| NCT02221310 | NYMC-515 L-10,349 | Ph 2 | completed | Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS |
| NCT02117297 | NYMC-504 L 10,394 | Ph 2 | completed | SCT Plus Immune Therapy in Average Risk AML/MDS |
| NCT03390296 results posted | 2017-0337 NCI-2018-00972, 2017-0337 | Ph 1, Ph 2 | completed | OX40, Venetoclax, Avelumab, Glasdegib, Gemtuzumab Ozogamicin, and Azacitidine in Relapsed or Refractory Acute Myeloid Leukemia |
| NCT03531918 results posted | 10000 NCI-2018-00776, 10000 | Ph 1, Ph 2 | completed | Gemtuzumab Ozogamicin With G-CSF, Cladribine, Cytarabine & Mitoxantrone for Untreated AML & High-Grade Myeloid Neoplasm |
| NCT04173585 TEAM | NCT-2017-0530 | Ph 2 | completed | TEAM-Trial: Targeting Epigenetic Therapy Resistance in AML With Bortezomib |
| NCT03878927 CPX GO | 1801018937 | Ph 1 | terminated | CPX-351+GO in Subjects 55 Years Old, or Older, With AML |
| NCT04326439 results posted | IRB00111627 | Ph 2 | terminated | AflacLL1901 (CHOA-AML) |
| NCT03727750 results posted | B1761031 2018-002619-89 | Ph 4 | completed | Evaluating QTc, PK, Safety of Gemtuzumab Ozogamicin (GO) in Patients With CD33+ R/R AML |
| NCT02724163 Myechild01 | RG_14-088 2014-005066-30 | Ph 3 | recruiting | International Randomised Phase III Clinical Trial in Children With Acute Myeloid Leukaemia |
| NCT01698879 results posted | ICOG-07 2007-006295-11 | Ph 2 | completed | Prospective Study of Mylotarg and G-CSF in Acute Myeloid Leukemia Treatment |
| NCT00895934 results posted | NCI-2012-01147 NCI-2012-01147, IR-6921 | Ph 1, Ph 2 | completed | Vorinostat, Azacitidine, and Gemtuzumab Ozogamicin for Older Patients With Relapsed or Refractory AML |
| NCT01548911 | IRB00019491 NCI-2012-00127, CCCWFU 22311 | Ph 2 | withdrawn | Gemtuzumab Ozogamicin in Treating Patients With Acute Myeloid Leukemia |
| NCT00673153 results posted | 2200.00 NCI-2010-00401 | Ph 2 | terminated | Vorinostat and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia |
| NCT00660036 | 07-154 | Ph 1 | terminated | Phase I/II Study of Mitoxantrone, Etoposide and Gemtuzumab Ozogamicin for Acute Myeloid Leukemia |
| NCT00798213 | P04717 | Ph 2 | terminated | SCH 727965 in Patients With Acute Myelogenous Leukemia and Acute Lymphoblastic Leukemia (P04717AM2)(TERMINATED) |
| NCT00882102 results posted | 2008-0288 | Ph 2 | completed | Decitabine and Gemtuzumab Ozogamicin in Acute Myelogenous Leukemia (AML) and High-Risk Myelodysplastic Syndrome (H-R MDS) |
| NCT00968071 results posted | 2007-0882 | Ph 2 | completed | Decitabine and Gemtuzumab Ozogamicin in Acute Myelogenous Leukemia and High-risk Myelodysplastic Syndrome |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
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
Indications & Usage
FDA Label (PDF)MYLOTARG is indicated for the treatment of Acute Myeloid Leukemia.
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.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment