With trial win for oral thalassemia drug, Agios charts broader path than gene therapy rivals

Gene therapies have been approved for certain patients with beta thalassemia. After a successful phase 3 readout, Agios hopes it can reach a unique subgroup and eventually all patients.

Agios’ mitapivat significantly increased blood hemoglobin levels in more patients than placebo did in patients with either alpha- or beta-thalassemia that were not dependent on transfusion, Agios said Wednesday.