FDA Approves First Oral Treatment for Anemia in Thalassemia, an Inherited Blood Disorder

https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/216196s003lbl.pdf?utm_medium=email&utm_source=govdelivery FDA Approves First Oral Treatment for Anemia in Thalassemia, an Inherited Blood Disorder Last week, the U.S. Food and Drug Administration (FDA) approved Aqvesme (mitapivat) tablets to treat anemia (low levels of red blood cells) in adults with alpha- or beta-thalassemia. This is the first oral treatment option for patients with beta-thalassemia and the first drug approval for patients with alpha-thalassemia. Disease or Condition Thalassemia is an inherited blood disorder caused by mutations in the genes responsible for producing alpha- and beta-globins. These globins are components of hemoglobin, a protein in red blood cells that carries oxygen. The abnormal effects on globin chain production damages the red blood cells, reducing their production and increasing their destruction (hemolysis). Some patients with thalassemia may be transfusion-dependent, requiring regular blood transfusions to manage their condition, typically every two to five weeks. Data Supporting Aqvesme The efficacy and safety of Aqvesme were evaluated in two multinational, randomized, double-blind, placebo-controlled clinical studies. The ENERGIZE-T study evaluated 258 adults with transfusion-dependent thalassemia, with 171 adults receiving Aqvesme and 87 receiving placebo. Efficacy was based upon transfusion reduction response, defined as greater than 50% reduction in the number of red blood cell units transfused with a reduction of at least two units in any consecutive 12-week period between the baseline visit and Week 48. A higher proportion of patients taking Aqvesme achieved a transfusion reduction response (30%) compared with the placebo group (13%). A second trial, the ENERGIZE study, evaluated Aqvesme in 194 adults with non-transfusion-dependent thalassemia over 24 weeks, with 130 adults receiving Aqvesme daily and 64 receiving placebo. Efficacy was based upon hemoglobin response (a measure of the improvement in anemia), defined as a ≥1 g/dL increase from baseline in mean hemoglobin concentration at Week 24. A higher proportion of patients taking Aqvesme achieved a hemoglobin response (42%) compared with the placebo group (2%). Another efficacy endpoint in the ENERGIZE study assessed the mean change from baseline in fatigue-related symptoms and impacts using a patient-reported outcome instrument, the Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue). The FACIT-F total score has a range of 0 to 52, with higher scores indicating less fatigue. At baseline, the mean FACIT-F score was approximately 36. Patients treated with Aqvesme had a mean increase in the FACIT-F total score of 4.9 compared to a mean increase of 1.5 in patients taking placebo. The recommended dosage for Aqvesme is 100 mg orally twice daily with or without food. Safety Information Aqvesme is only available through a restricted program called the Aqvesme Risk Evaluation and Mitigation Strategies (REMS) because of the risk of liver toxicity observed in the clinical trials. The most common side effects of Aqvesme are headache and insomnia. More safety information is available in the prescribing information.