FDA Approval: Gemtuzumab ozogamicin for the treatment of adults with newly-diagnosed CD33-positive acute myeloid leukemia

On September 1, 2017, FDA granted approval for gemtuzumab ozogamicin (GO) (Mylotarg; Pfizer, Inc) in combination with daunorubicin and cytarabine (DA) and as a monotherapy for the treatment of adult patients with newly-diagnosed CD33-positive acute myeloid leukemia (AML). GO is a CD33-targeted antibody-drug conjugate joined to calicheamicin. Approval of GO combination treatment was based on a randomized trial of 271 patients with newly-diagnosed AML treated with DA with or without 3 mg/m2 fractionated GO, which resulted in an event-free survival (EFS) of 13.6 months for GO + DA and 8.8 months for DA alone (HR 0.68 [95% CI: 0.51-0.91]). Hemorrhage, prolonged thrombocytopenia, and veno-occlusive disease were serious toxicities that were more common in patients treated with GO + DA. Approval of GO monotherapy was based on a randomized trial of 237 patients with newly-diagnosed AML treated without curative intent. Median overall survival (OS) was 4.9 months with GO versus 3.6 months on best supportive care (HR 0.69, [95% CI 0.53-0.90]). Adverse events were similar on both arms. Post-approval, several studies are required including evaluation of fractionated GO pharmacokinetics, safety of combination GO in the pediatric population, of immunogenicity, and of the effects of GO on platelet function.

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