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Mylotarg (gemtuzumab-ozogamicin)
Mylotarg works by binding to CD33 on cancer cells and releasing a toxic compound that kills the cells.
Mylotarg (gemtuzumab-ozogamicin) is a CD33-directed immunoconjugate originally developed by Wyeth Pharms Inc and currently owned by the same company. It targets the myeloid cell surface antigen CD33 and is used to treat acute myeloid leukemia. Mylotarg is a small molecule modality that was FDA-approved in 2000. The commercial status of Mylotarg is patented, and it is used to treat acute myeloid leukemia. Key safety considerations include potential side effects such as myelosuppression and infusion-related reactions.
At a glance
| Generic name | gemtuzumab-ozogamicin |
|---|---|
| Sponsor | Pfizer |
| Drug class | CD33-directed Immunoconjugate [EPC] |
| Target | Myeloid cell surface antigen CD33 |
| Modality | Monoclonal antibody |
| Therapeutic area | Oncology |
| Phase | FDA-approved |
| First approval | 2000 |
| Annual revenue | 100 |
Mechanism of action
Gemtuzumab ozogamicin is CD33-directed antibody-drug conjugate (ADC). The antibody portion (hP67.6) recognizes human CD33 antigen. The small molecule, N-acetyl gamma calicheamicin, is cytotoxic agent that is covalently attached to the antibody via linker. Nonclinical data suggest that the anticancer activity of gemtuzumab ozogamicin is due to the binding of the ADC to CD33-expressing tumor cells, followed by internalization of the ADC-CD33 complex, and the intracellular release of N-acetyl gamma calicheamicin dimethyl hydrazide via hydrolytic cleavage of the linker. Activation of N-acetyl gamma calicheamicin dimethyl hydrazide induces double-strand DNA breaks, subsequently inducing cell cycle arrest and apoptotic cell death.
Approved indications
- Newly-diagnosed CD33-positive AML
- Relapsed or refractory CD33-positive AML
Boxed warnings
- 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 )
Common side effects
- Hemorrhage
- Infection
- Veno-occlusive liver disease
- Thrombocytopenia
- Multi-organ failure
- Anemia
- Hepatic failure
- Hypotension
- Fever
- Nausea
- Vomiting
- Constipation
Key clinical trials
- Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia (PHASE2,PHASE3)
- Azacitidine and Gemtuzumab Ozogamicin in Treating Older Patients With Previously Untreated Acute Myeloid Leukemia (PHASE2)
- MYELOMATCH: A Screening Study to Assign People With Myeloid Cancer to a Treatment Study or Standard of Care Treatment Within myeloMATCH (MyeloMATCH Screening Trial) (PHASE2)
- 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) (PHASE2)
- Fludarabine Phosphate, Cytarabine, Filgrastim-sndz, Gemtuzumab Ozogamicin, and Idarubicin Hydrochloride in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome (PHASE2)
- Geriatric Assessment & Genetic Profiling to Personalize Therapy in Older Adults With Acute Myeloid Leukemia (PHASE2)
- IMPACT-AML: A Randomized Pragmatic Clinical Trial for Relapsed or Refractory Acute Myeloid Leukemia. (PHASE3)
- A Phase Ia/Ib Trial of Revumenib Combined With Cytarabine, Daunorubicin, and Gemtuzumab Ozogamicin (GO) in Frontline and Relapsed /Refractory Pediatric Acute Leukemia Patients (PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
| Biologic Exclusivity |
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Mylotarg CI brief — competitive landscape report
- Mylotarg updates RSS · CI watch RSS
- Pfizer portfolio CI