FDA Extends Gemtuzumab Ozogamicin Indication for Pediatric Patients with AML

June 16, 2020

The FDA extended the indication of gemtuzumab ozogamicin (Mylotarg) for newly diagnosed CD33-positive acute myeloid leukemia to include pediatric patients who are 1 month and older.

The FDA has extended the indication of gemtuzumab ozogamicin (Mylotarg) for newly diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients who are 1 month and older.

The efficacy and safety of gemtuzumab ozogamicin in the pediatric population was supported by data from the AAML00531 study, which was a multicenter, randomized study of 1,063 patients aged 1 month and older with newly diagnosed AML. Study participants were randomized to 5-cycle chemotherapy alone or with gemtuzumab ozogamicin (3 mg/m2) administered once on day 6 in induction 1 and once on day 7 in intensification 2.

The dual primary outcome measures were event-free survival (EFS), measured from the date of trial entry until induction failure, relapse, or death by any cause, and overall survival (OS). Secondary outcome measures included remission induction rate after 2 courses of induction therapy, disease-free survival, mortality, time to marrow recovery, and toxicities, including infectious complications.

The EFS hazard ratio was 0.84 (95% CI: 0.71-0.99). The estimated percentage of patients free of induction failure, relapse, or death at 5 years was 48% (95% CI, 43%-52%) in the gemtuzumab ozogamicin containing arm versus 40% (95% CI, 36%-45%) in the chemotherapy alone arm. No difference in OS was observed between the 2 arms.

The most common grade 3 and higher adverse reactions that occurred during induction 1 and intensification 2 in ≥5% of patients who received gemtuzumab ozogamicin were infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea, and hypotension.

Reference:

FDA. FDA approves gemtuzumab ozogamicin for CD33-positive AML in pediatric patients. FDA website. Published June 16, 2020. fda.gov/drugs/drug-approvals-and-databases/fda-approves-gemtuzumab-ozogamicin-cd33-positive-aml-pediatric-patients. Accessed June 16, 2020.