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 ...
People with the thalassemia trait, or thalassemia carriers, may not experience any symptoms. However, some thalassemia carriers can experience anemia symptoms, such as paleness and fatigue. In people ...
– Study Achieved the Primary Endpoint; Mitapivat Demonstrated a Statistically Significant Transfusion Reduction Response Compared to Placebo – – Statistical Significance Achieved for All Key Secondary ...
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