FDA approves Agios' Aqvesme for anemia in alpha and beta thalassemia, backed by Phase 3 data and a REMS safety program.

An oral pyruvate kinase (PK) activator, mitapivat becomes the only approved option for treating anemia in both ...

Approval was based on the phase 3 ENERGIZE and ENERGIZE-T studies evaluating the safety and efficacy of mitapivat in adults with alpha- or beta-thalassemia.

Agios Pharmaceuticals AGIO announced that the FDA has approved Aqvesme (mitapivat) for the treatment of adult patients with ...

The approval makes AQVESME the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, a rare blood disorder. The drug is expected ...

AQVESME is the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or ...

While gene therapy has emerged as a promising alternative for treating beta-thalassemia, hematopoietic stem cell transplantation (HSCT) remains the only widely accessible curative option for this ...

Our blood is a window to our health. Doctors examine biomarkers in our blood to determine our health status. These molecules can indicate if any abnormal processes are taking place, which could point ...

Agios Pharma’s oral PK activator, Aqvesme receives US FDA approval to treat anaemia in adults with alpha- or beta-thalassemia: Cambridge, Massachusetts Friday, December 26, 2025 ...

Investigators used MRI to evaluate brain perfusion in patients with transfusion-dependent (n=54) and non–transfusion-dependent (n=23) β-thalassemia versus 56 controls. They found that relative ...

After approving Casgevy (exagamglogene autotemcel) in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat ...

Agios Pharmaceuticals has announced a major regulatory milestone with the U.S. Food and Drug Administration approving ...