FDA Expands Mylotarg Approval to Include Children With Newly-Diagnosed CD33-Positive AML

June 16, 2020

The Food and Drug Administration (FDA) extended the indication of Mylotarg (gemtuzumab ozogamicin) to include the treatment of children aged one month or older with newly diagnosed CD33-positive acute myeloid leukemia (AML).

The Food and Drug Administration (FDA) extended the indication of Mylotarg (gemtuzumab ozogamicin) to include the treatment of children aged one month or older with newly diagnosed CD33-positive acute myeloid leukemia (AML).

The FDA, previously in 2017, approved the drug for the treatment of adults with newly diagnosed CD33-positive AML, as well as children aged two years and older with CD33-positive relapsed/refractory AML.

The agency based its latest decision on data from the multicenter, randomized AAML0531 trial which comprised 1,063 patients with newly diagnosed AML aged 0 to 29 years. During the trial, patients either received a five-cycle chemotherapy alone or with Mylotarg.

Approximately 48% of patients who received Mylotarg and chemotherapy did not experience induction failure, relapse, or death at five years, compared to 40% who received chemotherapy alone. However, there was no difference between the two arms regarding overall survival.

Common severe side effects that occurred in more than 5% of patients who received Mylotarg included — but were not limited to — decreased appetite, diarrhea, nausea and hypotension.

Check back later for you what you need to know about this approval.


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