FDA expands Mylotarg approval for pediatric AML subset
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.
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.