On June 16, 2020, the Food and Drug Administration extended the indication of gemtuzumab ozogamicin (MYLOTARG, Wyeth Pharmaceuticals LLC) for newly-diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients 1 month and older.
Efficacy and safety in the pediatric population were supported by data from AAML0531 (NCT00372593), a multicenter randomized study of 1,063 patients with newly-diagnosed AML ages 0 to 29 years. Patients 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 main efficacy outcome measure was event-free survival (EFS) measured from the date of trial entry until 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 five years was 48% (95% CI: 43%-52%) in the gemtuzumab ozogamicin + chemotherapy arm versus 40% (95% CI: 36%‑45%) in the chemotherapy alone arm. No difference between treatment arms in overall survival was demonstrated.
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.
This application was granted priority review. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
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