Agios Seeks FDA Nod to Expand Mitapivat Label in Sickle Cell Disease

Agios Pharmaceuticals AGIO announced that it has submitted a supplemental new drug application (sNDA) to the FDA seeking accelerated approval for the label expansion of its lead drug, mitapivat, for the treatment of sickle cell disease (SCD). Agios expects to receive notice of the sNDA filing acceptance and anticipated review timeline in the third quarter of 2026, following the authority’s 60-day filing review period.

Mitapivat, an oral pyruvate kinase (PK) activator, is marketed under two brand names in the United States — Pyrukynd and Aqvesme. While Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with PK deficiency, Aqvesme is approved for the treatment of anemia in adults with alpha- or beta-thalassemia. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.