Agios Pharmaceuticals' thalassemia drug meets the goals of late-stage study

In Phase 3 of ENERGIZE-T, Agios Pharmaceuticals’ drug mitapivat successfully boosted hemoglobin levels in some patients with thalassemia, bringing the company closer to potential approval for the first oral treatment for less severe forms of the disease. The drug met the main study goal by showing a statistically significant increase in hemoglobin response compared to placebo in non-transfusion-dependent alpha- and beta-thalassemia patients. Agios’ CEO Brian Goff stated that the company believes mitapivat has the potential to become the first approved oral therapy for non-transfusion-dependent thalassemia, with an estimated patient population of 8,000 in the U.S.

At OxyDial, we enthusiastically welcome the positive late-stage trial results for Agios Pharmaceuticals’ mitapivat in treating thalassemia patients. OxyDial fully supports making such transformative therapies available as potential new treatment choices for thalassemia patients.