Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency

Agios Pharmaceuticals, Inc. announced the submission of a New Drug Application (NDA) for mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults with pyruvate kinase (PK) deficiency, a rare, inherited disease leading to chronic hemolytic anemia. This condition accelerates the destruction of red blood cells, causing serious complications such as chronic fatigue, hemolytic crisis, gallstones, and more, significantly impacting patients' quality of life.

Chris Bowden, M.D., chief medical officer at Agios, emphasized the potential of mitapivat as the first disease-modifying therapy for PK deficiency, addressing the significant unmet needs of patients who currently rely on management strategies like blood transfusions and splenectomy, which carry their own risks.

The NDA submission is supported by data from two pivotal studies, ACTIVATE and ACTIVATE-T, which evaluated mitapivat in not regularly transfused and regularly transfused adults with PK deficiency, respectively. These studies, including patient-reported outcomes, were recently presented at the European Hematology Association (EHA) Virtual Congress. An ongoing extension study aims to assess the long-term safety, tolerability, and efficacy of mitapivat.

Mitapivat is a first-in-class pyruvate kinase R (PKR) activator, currently under evaluation for treating three distinct hemolytic anemias. Despite its potential, mitapivat has not yet been approved by any regulatory authority.

PK deficiency is associated with severe complications and quality of life challenges, with no currently approved therapies available. Agios, in partnership with PerkinElmer Genomics, has initiated the Anemia ID program to offer no-cost genetic testing for suspected hereditary anemias, including PK deficiency, aiming to improve diagnosis and disease management decisions.

This development marks a significant step forward in addressing the complex needs of PK deficiency patients, offering hope for a new therapeutic option that could modify the disease course and improve patient outcomes.