Agios Pharmaceuticals, Inc. (AGIO) announced on Wednesday that the Food and Drug Administration (FDA) has granted orphan drug designation to its drug candidate, Tebapivat, for the treatment of myelodysplastic syndromes (MDS), a category of blood cancers.
The company successfully completed a Phase 2a study of Tebapivat in patients with lower-risk MDS last year. Currently, Agios is embarking on a Phase 2b study targeting the same patient group.
The orphan drug designation offers multiple advantages, including tax credits, waivers of certain FDA fees for clinical trials, and the potential for seven years of market exclusivity upon the drug's approval.
