Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia

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[{"type":"text","content":"CAMBRIDGE, Mass., June 08, 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 thalassemia. Mitapivat is an investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated PKR enzymes.\n “Receiving orphan drug designation is an important milestone as we continue to advance mitapivat for patients with thalassemia, a serious hemolytic anemia with limited treatment options,” said Chris Bowden, M.D., chief medical officer at Agios. “We look forward to presenting updated data from our Phase 2 study of mitapivat in both alpha- and beta-thalassemia patients at the virtual European Hematology Association Annual Congress later this week.” The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials. Mitapivat was previously granted orphan drug designation by the FDA and the European Medicines Agency for pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anemia. Mitapivat Clinical Development Agios is conducting a Phase 2 study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of treatment with mitapivat in adults with non-transfusion-dependent β- and α-thalassemia (NTDT). The trial is fully enrolled, and the primary endpoint is hemoglobin response. Preliminary Phase 2 data establishing proof-of-concept for mitapivat in thalassemia were disclosed at the end of 2019, and updated data from this trial will be presented at the 25th European Hematology Association (EHA) Annual Congress, which is being held virtually on June 11-14, 2020. In addition, Agios has two ongoing global, pivotal trials in adults with PK deficiency that are fully enrolled. ACTIVATE: A placebo-controlled trial with a 1:1 randomization evaluating patients ...

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