Beta thalassemia
Jan 14, 2025
Agios’ PYRUKYND SNDA Accepted by FDA for Thalassemia; BridgeBio’s BBO-8520 Gets FDA Fast Track for KRASG12C-Mutated Lung Cancer; Nipocalimab Granted FDA Priority Review for Myasthenia Gravis; Rigel’s R289 Receives FDA Orphan Drug Designation for MDS; Rise Therapeutics Gets FDA IND Clearance for R-5780 in Cancer
FDA Accepts Agios’ SNDA for PYRUKYND in Thalassemia Patients Agios Pharmaceuticals, Inc. announced that the FDA has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) to treat adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The appl...
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Jan 21, 2022
Beta Thalassemia Treatment – Insights Into the Challenges & Solutions
Beta Thalassemia (β-Thalassemia) is a blood disorder characterized by reduced synthesis of the hemoglobin subunit beta (hemoglobin beta chain) that results in microcytic hypochromic anemia, an abnormal peripheral blood smear with nucleated red blood cells, and reduced amounts of hemoglobin A (HbA) on hemoglobin ana...
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