Agios strategy shift pays off with FDA approval of first drug for rare ...
Agios Pharmaceuticals shifted focus to rare genetic diseases, leading to FDA approval of Pyrukynd, the first treatment for pyruvate kinase deficiency anemia. Pyrukynd, a twice-daily tablet, showed efficacy in Phase 3 trials, offering a new option for patients previously reliant on blood transfusions. Agios plans further trials in children and other anemia types, maintaining Pyrukynd's price for five years.
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Agios Pharmaceuticals shifted focus to rare genetic diseases, leading to FDA approval of Pyrukynd, the first treatment for pyruvate kinase deficiency anemia. Pyrukynd, a twice-daily tablet, showed efficacy in Phase 3 trials, offering a new option for patients previously reliant on blood transfusions. Agios plans further trials in children and other anemia types, maintaining Pyrukynd's price for five years.