Cambridge, Massachusetts | December 26, 2025
Agios Pharmaceuticals announced a major regulatory milestone after the U.S. Food and Drug Administration (FDA) approved the expanded use of its drug mitapivat, marketed as Aqvesme, for the treatment of anemia in patients with alpha- or beta-thalassemia. The decision marks the first FDA approval of an oral therapy for both transfusion-dependent and non-transfusion-dependent forms of the disease.
Following the announcement, Agios shares rose sharply, reflecting strong investor confidence in the commercial and clinical potential of the expanded indication.
Thalassemia is a rare inherited blood disorder that impairs the body’s ability to produce healthy hemoglobin and red blood cells, often requiring lifelong management and regular transfusions. The FDA’s approval significantly broadens treatment options for adult patients, addressing a substantial unmet medical need.
Agios stated that Aqvesme is expected to become commercially available in the U.S. by late January 2026, following the rollout of a required safety program. The drug will carry a boxed warning for hepatocellular injury, with mandatory liver function monitoring every four weeks during the first 24 weeks of treatment. The label also advises against use in patients with cirrhosis. Analysts noted that the safety requirements were largely in line with market expectations.
The annual list price for Aqvesme is approximately $425,000 per patient, representing a modest premium compared to Pyrukynd, Agios’ previously approved mitapivat product for pyruvate kinase deficiency, which launched at a lower wholesale acquisition cost. Mitapivat was originally approved by the FDA in 2022, establishing the foundation for the company’s growing rare disease franchise.
Industry analysts estimate that the expanded approval could unlock an additional $320 million in peak annual revenue, layered on top of existing mitapivat sales. Agios targets an estimated 6,000 adult thalassemia patients in the U.S., with roughly 4,000 patients expected to be eligible at launch.
The FDA decision was supported by results from a late-stage clinical trial in which patients treated with mitapivat demonstrated a statistically significant improvement in hemoglobin levels compared with placebo.
With this expanded approval, Agios strengthens its position in rare hematologic diseases and significantly broadens the commercial scope of its mitapivat franchise, setting the stage for sustained growth as it enters 2026.
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