(Reuters) -Agios Pharmaceuticals said on Monday its experimental treatment for an inherited blood disorder met the main goal in a late-stage study.
The drug candidate, mitapivat, significantly reduced the need for blood transfusion in adult patients with transfusion-dependent alpha—or beta-thalassemia.
Thalassemia is an inherited blood disorder that affects the body’s ability to produce haemoglobin and healthy red blood cells.
The company plans to submit a marketing application for mitapivat in alpha- or beta- thalassemia patients, regardless of transfusion needs, to the U.S. health regulator by the end of this year.
(Reporting by Mariam Sunny in Bengaluru; Editing by Krishna Chandra Eluri and Ravi Prakash Kumar)
