FDA Fast Tracks AG-221 to Treat AML

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Agios Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has designated AG-221, which is currently undergoing clinical trials, Fast Track status for the treatment of acute myelogenous leukemia (AML) with a positive isocitrate dehydrogenase-2 (IDH2) mutation.


AG-221 is an orally available, selective, potent inhibitor of the IDH2 mutation. Agios Pharmaceuticals initiated a phase 1 multicenter, open-label, dose escalation clinical study of AG-221 to evaluate the safety and tolerability of the drug. IDH2 is an important enzyme utilized during the Krebs cycle, but in many cancers, the enzyme appears to be mutated. By blocking this enzyme in cancer cells, the cancer cells would be destroyed.


The FDA designates certain drugs Fast Track status in order to speed up development and the review of new drugs so that they may be made available to patients sooner. The FDA reserves this designation for medications that treat serious or life-threatening diseases or to fill an unmet medical need.


AML is a type of cancer in which the bone marrow makes abnormal myeloblasts, erythrocytes, or thrombocytes. The disease is known for its rapid disease progression and is the most common form of acute leukemia in adults.

Chemotherapy-free treatment a possibility for leukemia/lymphoma
The FDA has granted Fast Track designation to AG-221, a first-in-classinhibitor, to treat AML.
The US Food and Drug Administration (FDA) has granted Fast Track designation to Agios Pharmaceuticals' AG-221, a first-in-class, oral, selective, potent IDH2 mutant inhibitor, to treat patients with acute myelogenous leukemia (AML) that harbor an isocitrate dehydrogenase-2 (IDH2) mutation.

Currently, AG-221 is being evaluated in a Phase I clinical trial in patients with advanced hematologic malignancies. Agios chief medical officer Dr Chris Bowden said the company believes this designation is an important recognition by the FDA of the nonclinical and clinical data reported to date and the potential for AG-221 to address a significant unmet need for patients diagnosed with AML.

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