This morning, the U.S. Food and Drug Administration (FDA) approved Pfizer’s Mylotarg (gemtuzumab ozogamicin) for the treatment of adults with newly diagnosed acute myeloid leukemia (AML).
Approval of the targeted therapy also includes the use in the treatment of patients aged 2 years and older with CD33-positive AML who have experienced a relapse or who have not responded to initial treatment (refractory).
AML is a rapidly-progressing cancer of the blood and bone marrow, characterized by the nearly instantaneous expansion of white blood cells. The growth obstructs the production of typical blood cells.
“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 in a press release
Mylotarg was originally approved for use in 2000 as a stand-alone treatment for older patients with CD33-positive AML who had experienced relapse, but was voluntarily withdrawn from the market after later tests confirmed safety concerns and clinical benefit was in question. Today’s approval is highlighted by a lower recommended dose, an altered schedule as a stand-alone or in combination with chemotherapy, and a completely different patient population.
“Mylotarg’s history underscores the importance of examining alternative dosing, scheduling, and administration of therapies for patients with cancer, especially in those who may be most vulnerable to the side effects of treatment.,” said Pazdur.
Per the National Cancer Institute of the National Institutes of Health, an estimated 21,380 people will be diagnosed with AML in 2017, and more than 10,000 will die because of it.
Four different clinical trials took place to evaluate the safety and efficacy of Mylotarg – 2 assessing it in combination with chemotherapy for adults, and 2 studying it as a stand-alone treatment. Across all studies, patients treated with Mylotarg went longer without complications than patients without it, and a greater number of patients experienced complete remission .
Common side effects of Mylotarg include fever, nausea, infection, vomiting, and bleeding, among others. The drug comes with a boxed warning including the mention of severe or fatal liver damage, including blockage of veins in the liver.
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