FDA okays AQVESME as first treatment for anemia in alpha- & beta-thalassemia

The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia

Agios Pharmaceuticals has announced that the US FDA has approved AQVESME (mitapivat), an oral pyruvate kinase activator, for the treatment of anemia in adults with alpha- or beta-thalassemia.  

The approval makes AQVESME the only FDA-approved therapy for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. 

“Thalassemia is a debilitating disease that demands lifelong management and vigilant monitoring for many life-threatening complications, such as blood clots, heart disease, and liver disease. Despite its severity, treatments have historically been limited, leaving some patients without any options,” said Hanny Al-Samkari, Associate Professor at Harvard Medical School, and an investigator for the mitapivat thalassemia Phase 3 clinical program.  

“The ENERGIZE and ENERGIZE-T Phase 3 trial results demonstrate that AQVESME can help address anemia, fatigue, and the need for regular transfusions – key challenges of the disease. Today’s FDA approval represents an important step forward for individuals with thalassemia.” 

The FDA’s decision is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials, which included 452 adults with non-transfusion-dependent and transfusion-dependent thalassemia. The trials met all primary and key secondary endpoints, showing that AQVESME significantly improves hemoglobin levels, reduces transfusion burden, and alleviates fatigue compared to placebo. 

“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,” said Brian Goff, CEO of Agios.  

“With this approval, AQVESME becomes the only medicine indicated for the treatment of anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. We are deeply grateful to the patients, caregivers, healthcare providers, and advocacy partners who participated in and supported our clinical trials and helped ensure that our efforts reflected their priorities. Our focus now is on ensuring a successful launch and delivering meaningful impact for the community.” 

“This year marks 100 years since thalassemia was first described in medical literature, and the progress we’ve made as a community is truly extraordinary,” said Ralph Colasanti, National President, Cooley’s Anemia Foundation.  

“Innovative medicines like AQVESME help make this progress possible, offering new hope to patients like me by helping to address the complex needs and challenges of this disease. This approval provides an important new treatment option for the thousands of adults living with thalassemia in the U.S., including those with non-transfusion-dependent thalassemia who previously had no approved alternatives.” 

Safety monitoring will remain a key focus. In the trials, five patients experienced adverse reactions suggestive of hepatocellular injury (HCI), with two requiring hospitalization. Reactions occurred within the first six months and improved after stopping the drug.