Agios Pharma shares jump as US FDA expands approval for blood disorder drug

Agios (Nasdaq: AGIO) announced U.S. FDA approval of AQVESME™ (mitapivat) for treatment of anemia in adults with alpha- or beta-thalassemia, covering both non-transfusion-dependent and transfusion-dependent patients.

The approval is based on the randomized ENERGIZE and ENERGIZE-T Phase 3 trials (total 452 patients) that met primary and key secondary endpoints, showing improvements in hemoglobin, fatigue, and transfusion burden. AQVESME will be available in the U.S. in late January 2026 after implementation of an FDA REMS requiring liver tests and education. Five patients experienced adverse reactions suggestive of hepatocellular injury, with two hospitalizations; liver tests improved after discontinuation.

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