FDA Approves First Oral Anemia Treatment for Thalassemia

The Food and Drug Administration approved the first oral anemia treatment for adult patients with alpha- and beta-thalassemia on Monday. Mitapivat (Aqvesme, Agios) is now the first oral option for beta-thalassemia and the first drug approved for alpha-thalassemia.

FDA based the approval on findings from two randomized, placebo-controlled clinical trials. In the ENERGIZE-T trial, 30% of patients prescribed mitapivat achieved a transfusion reduction response. This was defined as a reduction of more than 50% in the number of red blood cell units transfused, with at least a two-unit decrease over any consecutive 12-week period. By comparison, 13% of patients given placebo achieved this response.

In the ENERGIZE trial, 42% of patients on mitapivat achieved a hemoglobin response, defined as an increase of more than 1 g/dL from baseline in mean hemoglobin by 24 weeks; 2% of patients on placebo achieved this outcome.

Mitapivat is only available through a risk evaluation and mitigation strategy (REMS) due to the risk of liver toxicity observed in the clinical trials.