Agios Pharma NDA for Pyruvate Kinase Deficiency

Agios Pharmaceuticals has submitted an NDA for mitapivat for treating adults with pyruvate kinase (PK) deficiency. The company says there are no approved therapies for PK deficiency, and current treatments include blood transfusions and splenectomy, which 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, it says.

The submission is based on data from two pivotal studies, ACTIVATE and ACTIVATE-T that “demonstrated a sustained, statistically significant increase in hemoglobin in patients with PK deficiency who are not regularly transfused,” Agios says. For example, in Activate 40% of patients randomized to mitapivat achieved a hemoglobin response, compared to zero patients randomized to placebo.