Health
After decades in which treatment has largely meant a lifetime of blood transfusions, adults living with thalassaemia-related anaemia now have an oral alternative.
Health
The USFDA approved mitapivat, sold as Aqvesme, the first pill designed to treat anaemia in people with alpha or beta thalassaemia, a common inherited blood disorder.
Health
The condition is passed down through families, in which faulty genes limit the body’s ability to produce healthy haemoglobin.
Health
Unlike blood transfusions, which temporarily replace red blood cells, mitapivat works by improving red blood cell energy metabolism, helping them survive longer and function effectively.
Health
The approval was based on results from the global ENERGIZE and ENERGIZE-T Phase 3 trials, which enrolled 452 adult patients with alpha or beta thalassaemia.