FDA Accepts Agios' Drug Application for PYRUKYND® in Adult Thalassemia Patients

On January 8, 2025, Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a frontrunner in cellular metabolism and pyruvate kinase (PK) activation therapy for rare diseases, announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat). This application pertains to the treatment of adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The FDA has classified this application under the Standard review category, with a Prescription Drug User Fee Act (PDUFA) target date set for September 7, 2025.

Thalassemia is a rare hereditary blood disorder that results in chronic anemia, which can lead to severe complications such as organ damage and stroke. Currently, patients suffering from thalassemia have limited treatment options. Dr. Sarah Gheuens, the chief medical officer and head of R&D at Agios, shared the company's enthusiasm about working closely with the FDA to advance the review process of their application. Agios aims to make PYRUKYND, an oral medication that modifies the disease, accessible to thalassemia patients regardless of their specific genetic makeup or transfusion requirements.

The supplemental application is built on data from the ENERGIZE and ENERGIZE-T Phase 3 trials. These trials evaluated the efficacy of mitapivat in comparison to a placebo in adults with different types of thalassemia. The ENERGIZE trial results were disclosed at the European Hematology Association 2024 Hybrid Congress in June 2024, while the findings from the ENERGIZE-T trial were presented at the 66th American Society of Hematology Annual Meeting and Exposition in December 2024.

PYRUKYND is a pyruvate kinase activator specified for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency. Agios leads innovations in PK activation and is committed to creating and distributing transformative therapies for individuals with rare diseases. In the United States, Agios is marketing a pioneering pyruvate kinase activator that addresses PK deficiency in adults, marking the first disease-modifying treatment for this rare form of hemolytic anemia.

Agios draws from its profound scientific acumen in classical hematology and its pioneering role in cellular metabolism and rare hematologic diseases to advance its comprehensive clinical pipeline. This pipeline comprises investigational medicines targeting conditions such as alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndrome-associated anemia (MDS), and phenylketonuria (PKU). Besides its clinical ventures, Agios is progressing with a preclinical TMPRSS6 siRNA, envisioned as a potential treatment for polycythemia vera.

The company is steadfast in its mission to bring groundbreaking therapies to patients globally, leveraging its leadership and scientific expertise to innovate and address unmet medical needs. Through its ongoing research and development efforts, Agios aims to transform the therapeutic landscape for rare diseases, offering hope and improved outcomes for affected individuals.

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