Indication Expanded for Gemtuzumab Ozogamicin

The FDA has extended the indication of gemtuzumab ozogamicin to include pediatric patients aged 1 month or older with newly diagnosed CD33-positive acute myeloid leukemia (AML). The treatment was previously approved for adults with newly diagnosed CD33-positive AML and patients aged 2 years and older with relapsed/refractory disease.

Approval was supported by data from AAML0531, a multicenter randomized study of 1,063 pediatric and young adult patients (age <29 years) with newly diagnosed AML. Patients were randomized to receive chemotherapy with or without gemtuzumab ozogamicin, dosed at 3 mg/m2,√ā¬†administered twice throughout the course of induction therapy.

The 5-year event-free survival rate was 48% in the gemtuzumab ozogamicin cohort versus 40% among patients who received chemotherapy only (hazard ratio = 0.84; 95% CI 0.71-0.99). No significant difference in overall survival was demonstrated between the two treatment arms.

The most common AEs associated with gemtuzumab ozogamicin, occurring in more than 5% of patients, were infection, febrile neutropenia, decreased appetite, hyperglycemia, mucositis, hypoxia, hemorrhage, increased transaminase levels, diarrhea, nausea, and hypotension.

Sources: FDA press release, September 1, 2017; FDA press release, June 16, 2020.