By SPC News Staff
The FDA approved mitapivat (Pyrukynd, Agios) to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency, a rare genetic blood disorder that affects an estimated one in 20,000 people worldwide.
The FDA based its decision, in part, on the results from two studies: a randomized, double-blind clinical study of 80 adults with PK deficiency who did not receive regular blood transfusions, and a single-arm study of 27 adults with PK deficiency who received regular blood