Agios Pharma Files US Application For Mitapivat In PK Deficiency

Agios Pharmaceuticals Inc AGIO has submitted an FDA marketing application seeking approval for mitapivat to treat adults with pyruvate kinase (PK) deficiency.
The company says, if approved, mitapivat could be the first potentially disease-modifying therapy for PK deficiency.
The submission is based on two studies, ACTIVATE and ACTIVATE-T, conducted in not regularly transfused and regularly transfused adults with PK deficiency, respectively.
A complete analysis of these data was recently presented at the European Hematology Association Virtual Congress.
An extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T is ongoing and designed to evaluate the long-term safety, tolerability, and efficacy of treatment with mitapivat.
The company remains on track to submit a marketing application in the EU in mid-2021 for mitapivat in adults with PK deficiency.
Price Action: AGIO shares are up 0.86% at $61 on the last check Monday.