CAMBRIDGE, Mass., Nov. 12, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s first-in-class pyruvate kinase R (PKR) activator mitapivat for the treatment of patients with sickle cell disease. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.
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Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Sickle Cell Disease
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