June 16, 2020
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FDA expands Mylotarg approval for pediatric AML subset

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The FDA today expanded the approval of gemtuzumab ozogamicin to include children aged 1 month and older newly diagnosed with CD33-positive acute myeloid leukemia.

Gemtuzumab ozogamicin (Mylotarg; Pfizer, Wyeth Pharmaceuticals) is a recombinant, humanized anti-CD33 antibody-drug conjugate.

Photomicrograph of bone marrow aspirate showing myeloblasts of acute myeloid leukemia (AML), a cancer of white blood cells.
FDA expanded the approval of gemtuzumab ozogamicin to include children aged 1 month and older newly diagnosed with CD33-positive acute myeloid leukemia.

FDA assessed the efficacy and safety of the agent for children using data from the multicenter randomized AAML0531 trial, which included 1,063 patients aged 0 to 29 years with newly diagnosed AML.

Researchers randomly assigned patients to five cycles of chemotherapy alone or with 3 mg/m2 gemtuzumab ozogamicin on day 6 of the induction 1 cycle and on day 7 of the intensification 2 cycle.

EFS, which served as the study’s main efficacy outcome measure, appeared significantly longer among patients assigned gemtuzumab ozogamicin (HR = 0.84; 95% CI, 0.71-0.99).

Forty-eight percent (95% CI, 43-52) of patients assigned gemtuzumab ozogamicin plus chemotherapy remained free of induction failure, relapse or death at 5 years compared with 40% (95% CI, 36-45) of patients assigned chemotherapy alone.

Researchers reported no difference in OS between the groups.

The most common grade 3 or worse adverse events associated with gemtuzumab ozogamicin that occurred during the induction 1 and intensification 2 cycles included infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea and hypotension.

The FDA previously granted gemtuzumab ozogamicin priority review for this indication.