The Food and Drug Administration (FDA) has approved Mylotarg (gemtuzumab ozogamicin for injection; Pfizer) for the treatment of adults with newly diagnosed CD33-positive acute myeloid leukemia (AML). Mylotarg has also been approved for the treatment of relapse or refractory CD33-positive AML in adults and in pediatric patients 2 years of age and older.

In May 2000, Mylotarg had received accelerated approval for the treatment of older patients with CD33-positive AML who had experienced a relapse, however, it was voluntarily withdrawn from the market in 2010 after confirmatory trials failed to show benefits and revealed safety concerns. This new approval for Mylotarg includes a new patient population, a lower recommended dose, and a different treatment schedule.

“We are approving Mylotarg after a careful review of the new dosing regimen, which has shown that the benefits of this treatment outweigh the risk,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

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In patients with newly diagnosed CD33-positive AML, Mylotarg in combination with chemotherapy increased event-free survival compared to chemotherapy alone (17.3 months vs. 9.5 months, respectively). Mylotarg was evaluated as a stand-alone treatment in two separate trials, one involving newly diagnosed AML patients and one in patients who had experienced a relapse of disease. In the trial, patients with newly diagnosed AML who received Mylotarg survived longer than those who received best supportive care (4.9 months vs. 3.6 months, respectively). For those with previous relapse, treatment with Mylotarg resulted in 26% of patients achieving complete remission which lasted a median 11.6 months.

Mylotarg carries a boxed warning regarding the risk of hepatotoxicity, including severe or fatal hepatic veno-occlusive disease, also known as sinusoidal obstruction syndrome.

The product will be supplied in a carton containing one 4.5mg single-dose vial for reconstitution and dilution.

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