MYLOTARG functions as a targeted therapy by selectively binding to the CD33 antigen on acute myeloid leukemia cells, delivering a potent cytotoxic agent to induce cancer cell death.

Mechanism of Action and Clinical Utility of MYLOTARG in AML

MYLOTARG is a CD33-directed antibody-drug conjugate. The gemtuzumab antibody component specifically targets the CD33 antigen, which is expressed on the surface of myeloid leukemia cells in most AML patients. Upon binding to CD33, MYLOTARG is internalized by the cell, and the cytotoxic agent, ozogamicin (a calicheamicin derivative), is released. Ozogamicin causes double-strand DNA breaks, subsequently inducing cell cycle arrest and apoptotic cell death. This targeted delivery mechanism aims to maximize efficacy against malignant cells while limiting systemic exposure to the cytotoxic agent. Its clinical efficacy is established for newly-diagnosed and relapsed/refractory CD33-positive AML. Specific outcome percentages from clinical trials are not detailed in the provided prescribing information highlights.