Mylotarg (gemtuzumab ozogamicin)
An approved treatment for Acute Myeloid Leukemia.
The same compound appears under different names depending on the context. Here is how to identify Gemtuzumab ozogamicin wherever you encounter it, plus the key facts at a glance.
- Generic name
- Gemtuzumab ozogamicin
- Brand name
- Mylotarg
- Development code
- CMA-676
- Drug class
- CD33-directed antibody-drug conjugate
- Manufacturer
- Pfizer (Wyeth)
- How it's taken
- IV infusion over 2 hours, on days 1, 4 and 7 of induction (combination regimen)
An IV infusion that delivers a cell-killing toxin to leukemia cells carrying the CD33 marker, which most AML cells have. Approved for newly diagnosed CD33-positive AML in adults and children 1 month and older, with chemotherapy or alone, and for relapsed or refractory CD33-positive AML from age 2. First approved in 2000, withdrawn in 2010 and re-approved at a lower, split dose on September 1, 2017.
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Where Gemtuzumab ozogamicin fits
An add-on to 7+3 induction for CD33-positive AML, used most often in core binding factor and other favorable-risk disease, and a single-agent option for older adults who cannot have chemotherapy. It is not a targeted mutation drug, so it can be combined with FLT3 inhibitors in trials.
How Gemtuzumab ozogamicin works
CD33 is a surface marker on most AML blasts. The antibody part of Mylotarg binds CD33 and is pulled inside the cell, where the attached calicheamicin is released and breaks DNA strands, killing the cell[1]. Lower, fractionated doses given on days 1, 4 and 7 of induction proved safer than the original single large dose while still clearing leukemia.
Mechanism: Antibody-drug conjugate: an anti-CD33 antibody that carries calicheamicin, a DNA-breaking toxin, into leukemia cells
Side effects and safety
- Infusion-related reactions. Including anaphylaxis; premedicate with a corticosteroid, acetaminophen and diphenhydramine and monitor during and for at least 1 hour after the infusion.
- Hemorrhage. Severe and fatal bleeding from prolonged low platelets; platelet counts are checked frequently.
- QT prolongation. ECG and electrolytes in people with a history of QT prolongation or on QT-prolonging drugs.
- Embryo-fetal toxicity. Can harm an unborn baby; contraception during treatment and for 6 months after (women) or 3 months after (men).
- Liver tests (ALT, AST, bilirubin, alkaline phosphatase) before each dose and often afterward
- Platelet and blood counts
- Observation during and after each infusion
- Careful review of VOD risk before any later transplant
Mylotarg carries a boxed warning for liver toxicity, including severe or fatal veno-occlusive disease (sinusoidal obstruction syndrome), which occurred in 5% of adults in the ALFA-0701 trial and is more likely after a stem cell transplant or at higher doses[1]. Other warnings are infusion reactions including anaphylaxis (premedication with a steroid, acetaminophen and an antihistamine, and 1 hour of observation), severe or fatal bleeding from prolonged low platelets, QT prolongation and harm to an unborn baby[1].
The most common side effects (more than 15%) were bleeding, infection, fever, nausea, vomiting, constipation, headache, raised AST and ALT, rash, mouth sores, febrile neutropenia and decreased appetite[1].
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Gemtuzumab ozogamicin
Given as an IV infusion over 2 hours in hospital. For newly diagnosed de novo AML in adults, the combination dose is 3 mg/m2 (up to one 4.5 mg vial) on days 1, 4 and 7 of induction with daunorubicin and cytarabine, and 3 mg/m2 on day 1 of each of 2 consolidation cycles[1]. As a single agent in newly diagnosed AML, adults receive 6 mg/m2 on day 1 and 3 mg/m2 on day 8, then up to 8 continuation courses of 2 mg/m2 every 4 weeks.
For relapsed or refractory AML (age 2 and older), 3 mg/m2 on days 1, 4 and 7. Children's doses are by body surface area or weight[1]. Premedication is given before every dose.
Availability and cost
Only available as the brand-name product.
Help paying for Mylotarg
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Co-Pay Assistance for certain Pfizer injectable or infused oncology medicines: eligible commercially insured patients pay as little as $0 per treatment, with a maximum benefit of $4,000 to $25,000 per calendar year depending on the product and no income requirement. Not for Medicare, Medicaid, TRICARE, VA or state programs.
For: private insurance · source - Free medicine program
Pfizer Patient Assistance Program provides Pfizer medicines free to eligible patients who have difficulty affording their Pfizer Oncology medicine, including some people with Medicare.
For: no insurance, underinsured, Medicare · source - Insurance and case manager help
Pfizer Oncology Together Care Champions help with insurance, financial assistance options and day-to-day resources. Call 1-877-744-5675.
For: private insurance, Medicare, Medicaid, TRICARE, no insurance, underinsured · source
Good to know: The Pfizer Oncology Together page describes program tiers without naming Mylotarg; the Mylotarg-specific maximum is in the co-pay portal terms.
Access and eligibility
The leukemia must be CD33 positive. Newly diagnosed use covers adults and children from 1 month; relapsed or refractory use covers adults and children from 2 years. The ALFA-0701 benefit was seen in people with favorable or intermediate-risk cytogenetics, which is how many specialists choose patients for it.
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
ALFA-0701 (NCT00927498) randomized 271 adults aged 50 to 70 with newly diagnosed de novo AML to daunorubicin and cytarabine with or without Mylotarg 3 mg/m2 on days 1, 4 and 7. Median event-free survival was 17.3 months with Mylotarg against 9.5 months without (hazard ratio 0.56, 95% CI 0.42 to 0.76); overall survival was not significantly different[1].
AML-19 (NCT00091234) randomized 237 older or unfit adults to single-agent Mylotarg or best supportive care; median survival was 4.9 against 3.6 months (hazard ratio 0.69, 95% CI 0.53 to 0.90)[1]. MyloFrance-1, a single-arm study in first relapse, supports the relapsed indication, and the children's study AAML0531 supported extending the newly diagnosed indication to age 1 month in June 2020[1][2].
Development history
Mylotarg was the first antibody-drug conjugate approved by the FDA, in May 2000, under accelerated approval for older adults with relapsed CD33-positive AML. It was withdrawn from the US market in 2010 when a confirmatory trial did not confirm the benefit. Pfizer resubmitted it with the lower fractionated dose from the French ALFA-0701 trial, and the FDA approved it again on September 1, 2017, adding children from age 1 month in June 2020[2].
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Common questions about Gemtuzumab ozogamicin
▸What is Gemtuzumab ozogamicin (Mylotarg)?
An IV infusion that delivers a cell-killing toxin to leukemia cells carrying the CD33 marker, which most AML cells have. Approved for newly diagnosed CD33-positive AML in adults and children 1 month and older, with chemotherapy or alone, and for relapsed or refractory CD33-positive AML from age 2. First approved in 2000, withdrawn in 2010 and re-approved at a lower, split dose on September 1, 2017.
▸How does Gemtuzumab ozogamicin work?
CD33 is a surface marker on most AML blasts. The antibody part of Mylotarg binds CD33 and is pulled inside the cell, where the attached calicheamicin is released and breaks DNA strands, killing the cell[1]. Lower, fractionated doses given on days 1, 4 and 7 of induction proved safer than the original single large dose while still clearing leukemia.
▸What are the side effects of Gemtuzumab ozogamicin?
Mylotarg carries a boxed warning for liver toxicity, including severe or fatal veno-occlusive disease (sinusoidal obstruction syndrome), which occurred in 5% of adults in the ALFA-0701 trial and is more likely after a stem cell transplant or at higher doses[1]. Other warnings are infusion reactions including anaphylaxis (premedication with a steroid, acetaminophen and an antihistamine, and 1 hour of observation), severe or fatal bleeding from prolonged low platelets, QT prolongation and harm to an unborn baby[1]. The most common side effects (more than 15%) were bleeding, infection, fever, nausea, vomiting, constipation, headache, raised AST and ALT, rash, mouth sores, febrile neutropenia and decreased appetite[1].
▸How is Gemtuzumab ozogamicin taken?
Given as an IV infusion over 2 hours in hospital. For newly diagnosed de novo AML in adults, the combination dose is 3 mg/m2 (up to one 4.5 mg vial) on days 1, 4 and 7 of induction with daunorubicin and cytarabine, and 3 mg/m2 on day 1 of each of 2 consolidation cycles[1]. As a single agent in newly diagnosed AML, adults receive 6 mg/m2 on day 1 and 3 mg/m2 on day 8, then up to 8 continuation courses of 2 mg/m2 every 4 weeks. For relapsed or refractory AML (age 2 and older), 3 mg/m2 on days 1, 4 and 7. Children's doses are by body surface area or weight[1]. Premedication is given before every dose.
▸Is Gemtuzumab ozogamicin FDA approved?
Yes, Gemtuzumab ozogamicin (Mylotarg) is FDA approved (2017) for the treatment of Acute Myeloid Leukemia.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- Wyeth Pharmaceuticals (Pfizer) via openFDA · 2025-12-19. Mylotarg (gemtuzumab ozogamicin) for injection prescribing information. https://api.fda.gov/drug/label.json?search=set_id:%2232fd2bb2-1cfa-4250-feb8-d7956c794e05%22
- FDA. Mylotarg approval history. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761060
- Pfizer. Financial assistance. https://www.pfizeroncologytogether.com/patient/financial-assistance