Agios Pharmaceuticals announced that the U.S. Food and Drug Administration approved its oral pyruvate kinase activator, AQVESME (mitapivat), for the treatment of anemia in adults with alpha‑ or beta‑thalassemia. The approval, granted on December 23, 2025, makes AQVESME the only FDA‑approved oral therapy for both transfusion‑dependent and non‑transfusion‑dependent thalassemia patients, a first‑in‑class breakthrough that addresses a long‑standing unmet need.
The approval is accompanied by a mandatory Risk Evaluation and Mitigation Strategy (REMS) program designed to monitor hepatocellular injury, a side effect observed in a small portion of patients during clinical trials. Under the REMS, patients must undergo liver function testing before starting therapy and every four weeks for the first 24 weeks, ensuring early detection of liver abnormalities.
Agios plans to launch AQVESME in the United States in late January 2026, following the implementation of the REMS program. The U.S. thalassemia market is estimated to include several thousand patients, while the global market is projected to reach $7.93 billion by 2030, underscoring the commercial potential of the new indication.
Financially, Agios reported a net loss of $96.5 million for Q4 2024, slightly higher than the $95.9 million loss in Q4 2023. Product revenue from its PK‑deficiency drug PYRUKYND reached $10.7 million in Q4 2024, a 51 % year‑over‑year increase from $7.1 million in Q4 2023, reflecting growing uptake of its existing therapy. The company also maintained a strong cash position of approximately $1.5 billion as of December 31 2024, providing a solid financial foundation for the new launch.
The approval expands Agios’s rare‑disease portfolio and positions the company as a key player in hematology. By offering the first oral therapy for both transfusion‑dependent and non‑transfusion‑dependent thalassemia, Agios gains a competitive advantage over existing treatments such as blood transfusions, iron chelation, and gene therapies. The new indication is expected to broaden the company’s revenue base and accelerate commercialization plans, potentially offsetting the modest net losses seen in recent quarters.
CEO Brian Goff emphasized the significance of the approval, stating, "Today is a landmark moment for the thalassemia community, bringing forward an innovative, disease‑modifying oral medicine to address the urgent needs of people living with this devastating rare blood disorder." He also noted that the REMS program, while adding a layer of monitoring, is a necessary step to ensure patient safety and long‑term efficacy.
Investors responded positively to the announcement, reflecting confidence in Agios’s ability to navigate the regulatory landscape and capitalize on a sizable unmet market. The approval is expected to strengthen the company’s market position and provide a foundation for future growth in the rare‑disease sector.
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