The sNDA is supported by data from the phase 3 ENERGIZE and ENERGIZE-T studies, which evaluated mitapivat vs placebo in patients with NTD and TD alpha- or beta-thalassemia, respectively.

There are two different families of thalassemia, which comes from the Greek word for sea. (Thalassemias are very common in ...

Beta thalassemia is a blood disorder that affects the production of hemoglobin. Hemoglobin is the iron-rich protein in your ...

Agios Pharmaceuticals, Inc. (AGIO), on Wednesday, announced that its supplemental New Drug Application seeking approval for the ...

FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and ...

Opens in a new tab or window Share on LinkedIn. Opens in a new tab or window Patients with beta-thalassemia major (also known as Cooley's anemia) have a reduction or absence of beta protein in ...

Agios Pharmaceuticals AGIO announced that the FDA has accepted its supplemental new drug application (sNDA) seeking label ...

Beta-thalassemia is a rare blood disorder caused by a genetic defect in hemoglobin. Several manufacturers are developing novel treatments for the disease, including Vertex, which has partnered ...

Considering taking supplements to treat beta thalassemia? Below is a list of common natural remedies used to treat or reduce the symptoms of beta thalassemia. Follow the links to read common uses ...