On June 16, 2020, the U.S. Food and Drug Administration (FDA) extended the indication of gemtuzumab ozogamicin (Mylotarg™) for newly-diagnosed CD33-positive acute myeloid leukemia (AML) to include pediatric patients aged 1 month and older.
Efficacy and safety in the pediatric population were supported by data from a multicenter randomized study (AAML0531; NCT00372593) of 1,063 patients with newly diagnosed AML aged 0–29. Patients were randomized to five-cycle chemotherapy alone or with gemtuzumab ozogamicin (3 mg/m2) administered once on day six in induction cycle one and once on day seven in intensification cycle two.
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 plus chemotherapy arm versus 40% (95% CI = 36%–45%) in the chemotherapy alone arm. The trial demonstrated no difference in overall survival between treatment arms.
The most common grade 3 and higher adverse reactions that occurred during induction cycle one and intensification cycle two (≥ 5%) were infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase, diarrhea, nausea, and hypotension.
FDA granted the application 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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