Agios Announces FDA Orphan Drug Designation for Tebapivat (AG-946) for the Treatment of Myelodysplastic Syndromes (MDS) Page 1

CAMBRIDGE, Mass., Sept. 11, 2024 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in breakthrough cellular metabolism and PK activation therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s novel pyruvate kinase (PK) activator tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS).

“Obtaining Orphan Drug designation for tebapivat in MDS underscores the importance of offering novel oral treatment options to patients suffering from this rare disease,” said Sarah Gheuens, MD, PhD, chief medical officer and head of R&D at Agios. “We aim to deliver the first oral therapy to address anemia due to ineffective erythropoiesis in lower-risk MDS, which affects approximately 75,000-80,000 patients in the US and EU5 and accounts for approximately 70% of MDS cases.”