AQVESME® (mitapivat), FDA-Approved for Adults with Thalassemia, Exclusively Available at Biologics by McKesson


Cary, N.C. , Dec. 29, 2025 – Biologics by McKesson, an independent specialty pharmacy specializing in oncology, cell and gene, and rare disease therapies, has been selected by Agios Pharmaceuticals, Inc. as the sole specialty pharmacy provider for AQVESME® (mitapivat), indicated for the treatment of adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.

Thalassemia is a rare, inherited blood disorder that causes chronic anemia and can lead to serious complications such as organ damage. Current treatment options are limited and include blood transfusions and iron chelation. This new indication builds on mitapivat’s initial approval in 2022 for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. Pyrukynd® (mitapivat) continues to be available for pyruvate kinase deficiency, while AQVESME® (mitapivat) introduces a new 100 mg strength for patients with thalassemia.

“Aqvesme represents a significant advancement for adults living with thalassemia, a lifelong condition with limited treatment options,” said George Paschal, vice president, business development, Biologics by McKesson. “We are proud to partner with Agios Pharmaceuticals as the exclusive specialty pharmacy provider for this important therapy and to help ensure timely, safe access for patients. Our team remains committed to delivering timely, safe access and high-touch support for patients and providers throughout the treatment journey.”

Because AQVESME® (mitapivat) is subject to a Risk Evaluation and Mitigation Strategy (REMS) program,  prescribers and pharmacies must comply with specific requirements to ensure safe use. At Biologics by McKesson, a dedicated REMS Care Team works directly with patients throughout their treatment journey—providing counseling, answering questions, and helping manage side effects to support adherence. This streamlined approach ensures patients receive their therapy as quickly as possible, minimizing delays and supporting continuity of care.

Biologics by McKesson is committed to and recognized for its level of customer service as well as its innovative, high-touch, and multidisciplinary approach. Putting patients first in all they do, the Biologics’ pharmacy team includes pharmacists with in-depth knowledge of therapies, experienced nurses, and financial counselors who are familiar with various financial assistance programs and organizations that help patients.

For the full prescribing information for AQVESME® (mitapivat), please visit https://www.aqvesme.com/thalassemia/

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