Gemtuzumab ozogamicin receives FDA-approval for CD33-positive acute myeloid leukemia in pediatrics

For newly diagnosed CD33-positive AML (acute myeloid leukemia), the FDA (Food and Drug Administration) extended the indication of gemtuzumab ozogamicin (MYLOTARG, Wyeth Pharmaceuticals LLC) on 16 June 2020 to include pediatric patients one month and older.

A multicentre randomized (AAML0531) study was conducted to explore the safety and efficacy of gemtuzumab ozogamicin in 1,063 patients with newly diagnosed AML aged 0 to 29 years.

Participants were randomized to either gemtuzumab ozogamicin (3mg/m²) administered once on day six in Induction 1 and once on day seven in Intensification 2 or 5-cycle chemotherapy alone.
Determination of EFS (Event-free survival) from the date of entry in the analysis until induction failure, relapse, or demise by any reason was the primary endpoint ascertained.

The EFS hazard ratio was found to be 0.84. The estimated percentage of patients free of induction failure, relapse, or death at five years was higher in the gemtuzumab ozogamicin plus chemotherapy group when compared to the group treated with chemotherapy alone as shown in the following table:

No difference between treatment arms in overall survival was demonstrated. Infection, reduced appetite, febrile neutropenia, mucositis, hyperglycemia, hypoxia, increased transaminase, hemorrhage, diarrhea, hypotension, and nausea were the most common grade 3 and higher adverse reactions that occurred during Induction 1 and Intensification 2 in ≥ 5% of patients receiving gemtuzumab ozogamicin.

Thus, in pediatric patients, gemtuzumab ozogamicin is safe and effective for managing CD33-positive AML.

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