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.

FDA Extends Indication for Gemtuzumab Ozogamicin for CD33-Positive AML in Pediatric Patients

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.

View full prescribing information for gemtuzumab ozogamicin.

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.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.

For assistance with single-patient oncology investigational new drug applications, contact the Oncology Center of Excellence's Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.