Agios Pharmaceuticals' blood disorder drug succeeds in late-stage study

[June 03, 2024]  (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)

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