4 September 2025 - Agios Pharmaceuticals today announced that the US FDA has extended the PDUFA goal date for the supplemental new drug application of Pyrukynd (mitapivat), an oral pyruvate kinase activator, for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassaemia by three months to 7 December 2025.

Following a recent information request from the FDA, Agios submitted a proposed risk evaluation and mitigation strategy to mitigate the risk of hepatocellular injury that was described in the original Pyrukynd supplemental new drug application.

Read Agios Pharmaceuticals press release