FDA

FDA greenlights mitapivat for alpha- or beta-thalassemia

January 7, 2026

Drug: Aqvesme (mitapivat)

Indication: Treatment of anemia in adults with alpha- or beta-thalassemia

Pharmacology: Pyruvate kinase activator

Boxed warnings

HEPATOCELLULAR INJURY. See full prescribing information for complete boxed warning. Aqvesme can cause serious hepatocellular injury. Measure liver laboratory tests (ALT, AST, alkaline phosphatase, and total bilirubin with fractionation) at baseline and every 4 weeks for 24 weeks and then as clinically indicated. Avoid use of Aqvesme in patients with cirrhosis. Discontinue Aqvesme if hepatocellular injury is suspected. Aqvesme is available only through a restricted program called the AQVESME REMS.

Availability

The drug is available only through a restricted drug safety program, called AQVESME REMS, because of the risk of hepatocellular injury. Notable requirements include:

• Prescribers must be certified by enrolling in the REMS and completing training.
• Prescribers must counsel patients receiving Aqvesme about the risk of hepatocellular injury.
• Prescribers must monitor liver tests (including ALT, AST, alkaline phosphatase, total bilirubin with fractionation, and other tests as clinically indicated) to determine if the patient is appropriate to receive Aqvesme treatment.
• Patients must enroll in the REMS and comply with the monitoring requirements.
• Pharmacies must be certified by enrolling in the REMS and must only dispense to patients who are authorized to receive Aqvesme.

Clinical studies

Alpha- and Beta-Thalassemia: A total of 301 patients with thalassemia received Aqvesme, administered at 100 mg orally twice daily, for up to 59.9 weeks in the ENERGIZE trial (N=129) and the ENERGIZE-T trial (N=172).

Source:

Aqvesme (mitapivat) [package insert]. Food and Drug Administration. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/216196s003lbl.pdf. Accessed January 5, 2025.