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The U.S. Food and Drug Administration has granted Fast Track designation to AG-221 for the treatment of patients with acute myelogenous leukemia (AML) that harbors an isocitrate dydrogenase-2 mutation. AG-221 is a first-in-class, oral, selective, potent IDH2 mutant inhibitor.

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The Fast Track designation, established under the FDA Modernization Act of 1997, is designed to facilitate frequent interactions with the FDA review team to expedite clinical development and submission of a New Drug Application for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs. The designation permits the drug developer the opportunity to submit sections of an NDA on a rolling basis as data become available, allowing the FDA to review those materials on a rolling basis as well.


This year, approximately 18,860 patients will be diagnosed with AML, and approximately 10,460 patients with AML will die, according to American Cancer Society statistics. A news release from the drug's manufacturer, Agios Pharmaceuticals, notes that approximately 20 percent of patients with AML have the IDH mutation and that


Phase I clinical trials for AG-221 are currently ongoing.