"With both our NDA and MAA filings, we are poised to deliver the first potentially disease-modifying therapy for people with PK deficiency, a chronic, lifelong hemolytic anemia characterized by serious complications affecting multiple organs," said Chris Bowden, M.D., chief medical officer at Agios. "There are no approved therapies for PK deficiency, and the current management strategies of blood transfusions and splenectomy are associated with both short- and long-term risks, including iron overload, blood clots and increased risk for infections. PK deficiency can lead to chronic fatigue, hemolytic crisis, gallstones, splenomegaly, liver cirrhosis, pulmonary hypertension and osteoporosis, and the burden of disease can take a toll on patients' ability to navigate work and other day-to-day activities, as well as on their mental health.