Agios Pharmaceuticals’ Mitapivat Shows Promise as Genetic Blood Disorder Treatment

Agios Pharmaceuticals has released positive phase 3 trial results for mitapivat, an investigational drug for treatment of adults with a rare genetic disorder, pyruvate kinase deficiency, who regularly receive blood transfusions.

Mitapivat decreased transfusions by 33 percent for 10 of the 27 patients enrolled in the study, following 24 weeks of treatment. Six of the 27 participants were transfusion-free during the same period.

Agios plans to file for FDA and European Medicines Agency (EMA) approval of mitapivat by mid-year.