FDA APPROVES AQVESME™ (mitapivat) | First Oral Therapy for Anaemia Across All Forms of Thalassaemia

This regulatory decision marks a landmark milestone in the management of thalassaemia, as AQVESME is now the first and only therapy approved in the United States indicated for treating anaemia in both non-transfusion-dependent (NTDT) and transfusion-dependent (TDT) forms of the disease.

Historically, treatment options have been limited, particularly for NTDT patients who do not require routine transfusions but still experience significant anemia and reduced quality of life. AQVESME’s approval therefore represents a significant advance in addressing unmet medical needs across the full spectrum of adult thalassaemia patients.

How Does AQVESME™ Work?

AQVESME™ is an oral activator of pyruvate kinase (PK), a key enzyme involved in red blood cell metabolism. By enhancing PK activity, the medicine helps improve red blood cell survival and energy balance, leading to increases in haemoglobin levels and reductions in symptoms such as fatigue. In addition, in patients with transfusion-dependent disease, AQVESME has been shown to reduce the transfusion burden. These effects were demonstrated in the global, randomised, double-blind Phase 3 clinical trials ENERGIZE (NTDT) and ENERGIZE-T (TDT), which enrolled a total of 452 adult patients representative of the real-world thalassaemia population and met all primary and key secondary efficacy endpoints.

Experts involved in the clinical programme have noted the importance of this approval for patients living with thalassaemia, emphasising that AQVESME may help address some of the key challenges of the disease, including chronic anemia, debilitating fatigue, and the complications associated with regular transfusions.

Availability and Expected Cost:

AQVESME™ is expected to become available in the U.S. in late January 2026, following implementation of the FDA-mandated Risk Evaluation and Mitigation Strategy (REMS) programme required to monitor liver safety due to observed rare cases of hepatocellular injury. Patients prescribed AQVESME will undergo liver function testing before starting treatment and at regular intervals thereafter as part of the REMS.

While this approval currently applies to the United States, Agios has also filed regulatory applications in other regions, including the European Union, where a positive scientific opinion has previously been issued, and final decisions are anticipated. Additional research is ongoing to explore the potential utility of mitapivat in other inherited haemoglobin disorders, such as sickle cell disease.

According to recent market reporting from financial news outlets and analyst commentary following the FDA approval of AQVESME™, the estimated annual cost of the therapy in the U.S. is approximately $425,000 per patient per year.

It should be noted that:

• This is an initial estimate based on early pricing hints shared by company executives and analysts, not an official published list price from Agios or the FDA.

• Actual out-of-pocket cost for patients will vary significantly depending on insurance coverage, patient assistance programmes, and healthcare system negotiation (e.g., Medicare, private insurers).

For more information on AQVESME™, please visit: https://www.aqvesme.com/thalassemia/

Sources:  Agios Pharmaceuticals / Reuters
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