Pyruvate kinase (PK) activation specialist Agios Pharmaceuticals Inc (Nasdaq:AGIO) announced on Wednesday that the US Food and Drug Administration (FDA) has granted orphan drug designation to tebapivat (AG-946), a novel PK activator, for the treatment of myelodysplastic syndromes (MDS).

This designation supports the development of tebapivat as a potential new treatment option for patients with this rare disease.

Agios completed a Phase 2a study of tebapivat in lower-risk MDS late last year and is currently initiating a Phase 2b study.

Mitapivat, the company's lead PK activator, was previously granted FDA orphan drug designation for the treatment of PK deficiency, thalassemia and sickle cell disease.