The Food and Drug Administration on Thursday approved a new drug from Agios Pharmaceuticals to treat people with a rare form of anemia.
The Agios drug, mitapivat, is the first approved treatment for pyruvate kinase deficiency, a rare, inherited disorder that results in the rapid destruction of red blood cells. Given as a pill, mitapivat is designed to activate an enzyme called pyruvate kinase-R that red blood cells use to convert sugars into energy.
PK deficiency affects approximately 3,000 people in the U.S.
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