FDA Approves Aqvesme as First Oral Therapy for Anemia in Alpha- and Beta-Thalassemia

Key facts

• Aqvesme (mitapivat) tablets are the first oral treatment approved for anemia in adults with alpha- or beta-thalassemia.
• In the ENERGIZE-T study of transfusion-dependent thalassemia, 30% of Aqvesme-treated patients achieved >50% transfusion reduction versus 13% on placebo.
• In the ENERGIZE study of non-transfusion-dependent thalassemia, 42% of Aqvesme recipients achieved ≥1 g/dL hemoglobin rise at Week 24 versus 2% on placebo.
• Aqvesme-treated patients reported a mean 4.9-point improvement in FACIT-Fatigue score versus 1.5 points with placebo.
• Aqvesme is available only through a REMS program due to observed liver toxicity risk.