FDA just approved the 1st-ever oral drug for thalassemia anemia
Big news for people with thalassemia: the FDA has greenlit Aqvesme (mitapivat), the first pill to treat anemia in adults with both alpha and beta-thalassemia.
Until now, many patients, especially those with transfusion-dependent thalassemia, had to rely on regular blood transfusions—so this could be a real game-changer for about 6,000 folks in the US.
How does Aqvesme actually help?
Aqvesme is taken twice a day and works by boosting an enzyme in red blood cells.
In clinical trials, nearly half of patients who didn't need transfusions saw their hemoglobin levels go up, while about a third of those who did need transfusions were able to cut their transfusion needs by more than half.
That's a big deal for anyone tired of endless hospital visits.
What even is thalassemia?
Thalassemia is a genetic disorder where your body can't make normal hemoglobin, so you don't get enough healthy red blood cells.
Many people with transfusion-dependent thalassemia need blood transfusions every few weeks plus meds to prevent iron overload—a tough routine that Aqvesme now hopes to make easier.