The Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adults with non-transfusion-dependent (NTD) and ...

Results showed 30.4% of patients treated with mitapivat achieved a transfusion reduction response vs 12.6% of patients who received placebo. Mitapivat was associated with a significant reduction in ...

Sickle cell thalassemia occurs when you inherit both a sickle cell gene mutation and a thalassemia gene mutation. Symptoms are similar to other types of sickle cell disease but can vary depending on ...

About PYRUKYND® (mitapivat) U.S. INDICATION PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

–Mitapivat Demonstrated a Statistically Significant Increase in Hemoglobin Response Rate Compared to Placebo – – Statistical Significance Also Achieved for Key Secondary Endpoints of Change From ...

Agios Pharmaceuticals said a key European regulatory committee has recommended expanded approval of its Pyrukynd anemia drug for certain people with the inherited blood disorder thalassemia. Agios on ...

– Study Achieved the Primary Endpoint; Mitapivat Demonstrated a Statistically Significant Transfusion Reduction Response Compared to Placebo – – Statistical Significance Achieved for All Key Secondary ...