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# Agios Planning NDA for Anemia Disorder Drug
- URL: https://www.fdaweb.com/agios-planning-nda-for-anemia-disorder-drug/
- Published: 2021-01-27T12:00:00.000Z
- Updated: 2026-09-14T16:44:18.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5148540

Agios Pharmaceuticals says it will file an NDA in the second quarter for mitapivat for treating adults with pyruvate kinase (PK) deficiency following an announcement of positivePhase 3 data from the ACTIVATE-T trial. The data show that mitapivat in regularly transfused adults with PK deficiency demonstrated a statistically significant and clinically meaningful reduction in transfusion burden. Agios [recently reported](https://www.globenewswire.com/Tracker?data=lQpaB%5FObjWoE6S31FRYENKS8rPvjL4b7ONGb7yolEWlC%5FqitWbMFUbd0l5Vdyb662L2cyjFFUtZdoTQ13tj5MihnmmAoGebHo-jF7sxkwWnTIw5RzW2CgEL4Y4oMfNayUZjfP31kXF6sdlyGkeYxJgWLXpQo1Lsn7LXXqU2nI5tb%5FAmJfaBBZXak26Bv2WNSWWtX5PXdJ6TGNuTeFBU3MNj891BUm1e4SYm4%5Fd6MiTM=&ref=fdaweb.com) that its Phase 3 ACTIVATE trial of mitapivat in adults with PK deficiency who did not receive regular transfusions met its primary endpoint, with 40% of patients randomized to mitapivat achieving a hemoglobin response compared to 0 patients randomized to placebo.

PK deficiency is a rare, inherited disease that presents as chronic hemolytic anemia, which is the accelerated destruction of red blood cells, according to the company. It is associated with serious complications, including gallstones, pulmonary hypertension, extramedullary hematopoiesis, osteoporosis and iron overload and its sequelae, which can occur regardless of the degree of anemia or transfusion burden.