Agios Pharmaceuticals Secures FDA Approval For AQVESME, Supported By Global Phase 3 Trials Showing Major Gains In Hemoglobin And Quality Of Life

Agios Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company developing innovative treatments for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has approved AQVESME™ (mitapivat). This oral pyruvate kinase (PK) activator is now authorized for treating anemia in adults with either alpha- or beta-thalassemia. With this approval, AQVESME becomes the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent forms of alpha- or beta-thalassemia.

Thalassemia is a serious inherited blood disorder that requires lifelong care and constant monitoring for complications such as heart disease, liver disease, and blood clots. According to Dr. Hanny Al-Samkari, an investigator in the Phase 3 clinical program, treatment options have long been limited, leaving many patients with no approved therapies. He noted that results from the ENERGIZE and ENERGIZE-T Phase 3 trials show that AQVESME can improve key symptoms and challenges of thalassemia, including anemia, fatigue, and dependence on blood transfusions. He emphasized that the FDA’s approval marks a major advancement for patients living with this condition.

The FDA’s decision is supported by data from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials, which enrolled 452 adults representing the real-world thalassemia population. The ENERGIZE trial evaluated patients with non-transfusion-dependent thalassemia, while ENERGIZE-T studied those who rely on regular blood transfusions. Both trials met all primary and key secondary endpoints, demonstrating that AQVESME significantly improves hemoglobin levels, reduces transfusion needs, and enhances measures of fatigue and overall quality of life compared to placebo.

Agios CEO Brian Goff highlighted that this approval marks a milestone for the thalassemia community. He noted that AQVESME is now the only medicine approved to treat anemia across both major patient groups—those who require transfusions and those who do not. He thanked the patients, healthcare teams, and advocacy organizations involved in the clinical development program, and emphasized that the company’s priority is to ensure a successful launch and deliver meaningful benefits to the community.

Ralph Colasanti, National President of the Cooley’s Anemia Foundation, added that this approval comes at a meaningful moment, as the medical community marks 100 years since thalassemia was first described. He explained that new treatments like AQVESME offer hope and address longstanding unmet needs, especially for patients with non-transfusion-dependent thalassemia who previously had no approved therapeutic options.

During the trials, five patients treated with AQVESME experienced signs of hepatocellular injury (HCI), and two of them required hospitalization. These adverse reactions occurred within the first six months of treatment and improved after the medication was discontinued. To manage this risk, the FDA has required a Risk Evaluation and Mitigation Strategy (REMS) program known as AQVESME REMS. The REMS program includes specific liver function monitoring—before starting treatment, every four weeks for the first 24 weeks, and then as needed. It also requires education and certification for patients, prescribers, and pharmacists.

Because of the REMS requirements, mitapivat will be sold under the brand name AQVESME in the U.S. for its thalassemia indication. For its previously approved indication in pyruvate kinase (PK) deficiency, the medicine will continue to be marketed under the name PYRUKYND® (mitapivat), which does not require a REMS program. Outside the United States, mitapivat will continue to be known as PYRUKYND for both PK deficiency and thalassemia in regions where it is already approved, and will keep this name in countries currently undergoing regulatory review.