Agios Pharmaceuticals announced that the FDA has granted Orphan Drug Designation for AG-221, an oral first-in-class IDH2 mutant inhibitor for the treatment of patients with acute myelogenous leukemia (AML).

AG-221 is currently being evaluated in a Phase 1 clinical trial in patients with advanced hematologic malignancies that carry an IDH2 mutation.

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AML prevalence is estimated to be approximately 35,000—40,000 patients in the U.S., with approximately 15% of patients estimated to carry an IDH2 mutation. The FDA grants Orphan Drug status to support development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S.

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