Drug Therapy

FDA Approves New Drug for AML Subtype

The US Food and Drug Administration (FDA) has approved Mylotarg as a treatment for adults with CD33-positive acute myeloid leukemia (AML) and for patients aged 2 years or older with CD33-positive AML who have experienced a relapse or who were refractory to initial treatment.
FDA Approves New Treatment Option for Poor-Prognosis AML
FDA Approves New Option for Acute Myeloid Leukemia

Mylotarg was originally approved in 2000 as a stand-alone therapy for older patients with CD33-positive AML who had experienced a relapse but was later taken off the markey due to suspect clinical benefit and safety concerns. The new approval includes a lower recommended dose, a different schedule in combination with chemotherapy or on its own, and a new patient population.

Serious adverse events associated with Mylotarg include low blood counts, infections, liver damage, hepatic veno-occlusive disease, infusion-related reactions, and hemorrhage.

Mylotarg comes with a boxed warning that severe or fatal liver damage—including veno-occlusive disease or sinusoidal obstruction syndrome—may occur.

Melissa Weiss


FDA approves Mylotarg for treatment of acute myeloid leukemia [press release]. US Food and Drug Administration. September 1, 2017. Accessed September 1, 2017.