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FDA approves Agios' Aqvesme for anemia in alpha and beta thalassemia, backed by Phase 3 data and a REMS safety program.
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Approval was based on the phase 3 ENERGIZE and ENERGIZE-T studies evaluating the safety and efficacy of mitapivat in adults with alpha- or beta-thalassemia.
Agios Pharmaceuticals shares surged after the FDA approved expanded use of its drug mitapivat for treating anemia in alpha- and beta-thalassemia patients.