New Drug Application (NDA) submitted to FDA for mitapivat for treatment of adults with Pyruvate Kinase Deficiency

NDA for first potential-disease-modifying therapy for people with this chronic, lifelong haemolytic anaemia chronic, usually managed with blood transfusions and splenectomy, is supported by data from ACTIVATE and ACTIVATE-T studies. EU regulatory submission planned for mid-2021.

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Biospace Inc.