Aqvesme (mitapivat)

The first oral pill that treats anemia in adults with alpha- or beta-thalassemia by boosting energy production inside red blood cells. By activating a key enzyme called pyruvate kinase, it helps red blood cells survive longer and function better, raising hemoglobin levels without transfusions.

Aqvesme has a boxed warning for serious liver injury (hepatocellular injury). In the thalassemia trials, 5 patients had liver injury within the first 6 months and 2 needed hospital care; it improved after the drug was stopped. Liver tests are required before starting, every 4 weeks for 24 weeks, and then as needed. People with cirrhosis should not take it. Report loss of appetite, nausea, pain in the upper right belly, vomiting, yellow skin or eyes, or dark urine. Because of this risk, Aqvesme is only available through the AQVESME REMS program. The most common side effects were headache and insomnia.

The recommended dose for thalassemia is 100 mg by mouth twice daily, with or without food. Swallow tablets whole; do not split, crush, chew, or dissolve them. Liver tests are needed before the first dose, every 4 weeks for the first 24 weeks, and then as your doctor decides. Aqvesme is only available through the AQVESME REMS program.

Yes, Mitapivat (Aqvesme) is FDA approved (2025) for the treatment of Thalassemia (alpha and beta).

Mitapivat is the first oral disease-modifying therapy for thalassemia. Rather than replacing missing hemoglobin or providing blood transfusions, it activates pyruvate kinase to boost energy production inside red blood cells, helping them survive longer and reducing anemia from within.

Due to the risk of liver enzyme elevations, mitapivat is only available through the AQVESME REMS (Risk Evaluation and Mitigation Strategy) program. This requires liver function monitoring before treatment, every 4 weeks for the first 24 weeks, and periodically thereafter.

Yes. Mitapivat was first approved in February 2022 under the brand name Pyrukynd for pyruvate kinase deficiency, a rare inherited anemia. The thalassemia indication under the brand name Aqvesme was approved in December 2025, expanding its use to a much larger patient population.

The ENERGIZE Phase 3 trial enrolled 194 patients with non-transfusion-dependent alpha or beta-thalassemia. 42% of patients on mitapivat achieved a hemoglobin increase of at least 1.0 g/dL sustained over weeks 12-24, compared to just 2% on placebo.

For thalassemia, Aqvesme is taken as 100 mg by mouth twice daily, with or without food. Tablets should be swallowed whole. (The lower, step-up doses of 5, 20 and 50 mg apply to Pyrukynd for pyruvate kinase deficiency, not to Aqvesme.)

Mitapivat can potentially be combined with other therapies, though clinical trial data primarily studied it as monotherapy. Its unique oral mechanism of action complements existing approaches like luspatercept or transfusion support. Discuss combination strategies with your hematologist.

Yes. The ENERGIZE trial included patients with both non-transfusion-dependent alpha and beta-thalassemia, and the drug showed benefit across both subtypes. This is significant because most thalassemia therapies are studied primarily in beta-thalassemia.

Not yet. Agios filed for accelerated approval in sickle cell disease, and the FDA granted priority review with a decision date of November 1, 2026^[2]. In the RISE UP Phase 3 trial of 207 people aged 16 and older, 40.6% on mitapivat had a hemoglobin response compared with 2.9% on placebo, but pain crises, 2.62 a year on mitapivat versus 3.05 on placebo, were not significantly reduced^[1].