FDA Grants Orphan Drug Status to Agios' Tebapivat for MDS Treatment

Agios Pharmaceuticals, Inc., located in Cambridge, Mass., has announced that the U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to its novel pyruvate kinase (PK) activator, tebapivat (AG-946), for the treatment of myelodysplastic syndromes (MDS). MDS is a rare condition affecting approximately 75,000-80,000 individuals in the U.S. and EU5 and accounts for about 70% of all MDS cases.

Sarah Gheuens, M.D., Ph.D., the chief medical officer and head of R&D at Agios, highlighted the significance of receiving this designation. She emphasized the importance of introducing new oral treatment options for patients with this rare disease. Tebapivat aims to address anemia caused by ineffective erythropoiesis in lower-risk MDS patients.

Agios has completed a Phase 2a study of tebapivat in lower-risk MDS patients and is moving forward with a Phase 2b study. The FDA's orphan drug designation is part of its initiative to support the development of treatments for rare disorders affecting fewer than 200,000 people in the United States. This designation provides companies with various incentives, including tax credits, exemptions from certain FDA fees related to clinical trials, and the potential for seven years of market exclusivity upon drug approval.

Agios’ primary PK activator, mitapivat, has also been granted orphan drug designation by the FDA for treating PK deficiency, thalassemia, and sickle cell disease. As of now, tebapivat is not approved for use by any regulatory authority.

Agios Pharmaceuticals is a leader in PK activation and focuses on developing groundbreaking therapies for patients with rare diseases. The company currently markets a first-in-class pyruvate kinase activator for adults with PK deficiency in the U.S., which is the first disease-modifying therapy for this rare, chronic, and debilitating hemolytic anemia.

Agios is leveraging its deep scientific expertise in classical hematology and leadership in cellular metabolism and rare hematologic diseases to advance a broad clinical pipeline. This includes investigational medicines targeting alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia, and phenylketonuria (PKU). Moreover, the company is progressing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera.