Agios Pharmaceuticals announces FDA granted ODD to AG-946

Agios Pharmaceuticals announced that the U.S. Food and Drug Administration, FDA, has granted orphan drug designation, ODD, to the company’s novel pyruvate kinase, PK, activator tebapivat, AG-946, for the treatment of myelodysplastic syndromes, MDS. “Receiving orphan drug designation for tebapivat in MDS underscores the importance of bringing new oral treatment options to patients suffering from this rare disease,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head R&D at Agios. “We aim to deliver the first oral therapy that addresses anemia due to ineffective erythropoiesis in lower-risk MDS, which affects approximately 75,000-80,000 patients in the U.S. and EU5 and accounts for approximately 70% of MDS cases.”