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FDA Approves Gemtuzumab Ozogamicin for Certain Patients With AML
On June 16, 2020, the FDA extended the approval of gemtuzumab ozogamicin (Mylotarg, Wyeth Pharmaceuticals) to include newly diagnosed CD33-positive acute myeloid leukemia (AML) in patients aged 1 month and older.
This approval is based on efficacy and safety results from the pediatric population of the multicenter, randomized AAML0531 trial.
The AAML0531 trial included 1063 patients with newly diagnosed AML aged 0 to 29 years. Patients were randomized to receive 5 cycles of chemotherapy alone or in combination with gemtuzumab ozogamicin administered at 3 mg/m2 once on day 6 during Induction 1 and once on day 7 during Intensification 2.
The main efficacy outcome measure was event-free survival (EFS), defined as the date from trial entry to induction failure, relapse, or death by any cause.
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 arm compared with 40% (95% CI: 36%-45%) in the chemotherapy alone arm. Overall survival was similar between the 2 treatment arms.
The most common grade ≥3 adverse events (≥5% patients) associated with gemtuzumab ozogamicin were infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea, and hypotension.—Janelle Bradley
Source: US Food and Drug Administration. FDA approves gemtuzumab ozogamicin for CD33-positive AML in pediatric patients. Updated June 16, 2020. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-gemtuzumab-ozogamicin-cd33-positive-aml-pediatric-patients. Accessed June 16, 2020.
