A major breakthrough for adults living with rare inherited blood disorders, the U.S. Food and Drug Administration (FDA) has officially approved Agios Pharmaceuticals’ oral drug AQVESME™ (mitapivat) for the treatment of anemia in both alpha- and beta-thalassemia, regardless of whether patients require regular blood transfusions or not.
Agios Pharmaceuticals, a biotechnology company focused on developing transformative therapies for rare diseases, announced late Tuesday that it has received FDA approval for AQVESME after years of clinical research and trial work. With this action, the drug becomes the first and only FDA-approved medicine for anemia in adults with both non-transfusion-dependent and transfusion-dependent forms of alpha- or beta-thalassemia.
The approval is based on results from two global Phase 3 clinical trials, ENERGIZE and ENERGIZE-T, which together enrolled hundreds of patients who represent the real world thalassemia population. In these studies, adults treated with AQVESME showed significant improvements in hemoglobin concentration, reduced need for blood transfusions, and improved quality-of-life measures such as reduced fatigue, compared with placebo groups.
“This is a landmark moment for the thalassemia community,” said Agios CEO Brian Goff in the company’s press release. He emphasized that many adults with this lifelong condition previously faced a severe lack of approved treatment options. With this approval, AQVESME offers a new, orally administered therapy that addresses key clinical challenges for patients.
Thalassemia is a genetic blood disorder that affects the body’s ability to produce healthy hemoglobin, the protein responsible for carrying oxygen in red blood cells, and can cause chronic anemia, debilitating fatigue, and severe complications such as heart and liver disease. Depending on the severity, some patients require frequent blood transfusions that can lead to iron overload and other long-term risks.
Despite the celebration surrounding the approval, the FDA is requiring a Risk Evaluation and Mitigation Strategy (REMS) program for AQVESME due to observed cases of potential liver injury in a small number of trial patients. Under the REMS, patients and healthcare providers must follow strict liver test monitoring protocols before and after starting treatment.
Agios expects the drug to be available in the U.S. in late January 2026, once the REMS program is implemented. The company also continues to market mitapivat under the brand name PYRUKYND® for other approved indications such as pyruvate kinase deficiency, but AQVESME will be the brand used specifically for thalassemia in the United States.
Patient advocacy groups and medical experts have praised the FDA’s decision as a crucial advance. Ralph Colasanti, National President of the Cooley’s Anemia Foundation, noted that this approval “provides an important new treatment option for the thousands of adults living with thalassemia in the U.S.,” especially for those who did not previously have any approved therapies.
With Agios’ shares responding positively in the market following the news, attention now turns to successful launch execution and real-world outcomes as patients and clinicians prepare for this new treatment era.
