Agios Submits sNDA to FDA for U.S. Accelerated Approval of Mitapivat in Sickle Cell Disease

CAMBRIDGE, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the U.S. accelerated approval of mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease. The submission follows agreement with the FDA on the confirmatory clinical trial, which is required under the accelerated approval pathway.

The confirmatory clinical trial is designed to demonstrate the clinical benefit of mitapivat on reducing transfusion burden in sickle cell disease, with a primary endpoint of transfusion free from Week 4 through Week 52. The global, randomized, double-blind, placebo-controlled, 52-week trial will enroll approximately 159 patients aged 12 years or older with sickle cell disease. The design of the confirmatory trial is informed by a clinically meaningful reduction in transfusion burden that was observed with mitapivat compared to placebo in the RISE UP Phase 3 trial based on data systematically and prospectively collected. These results will be included in an oral presentation on the RISE UP Phase 3 trial during the Plenary Abstracts Session at the 31st European Hematology Association (EHA) Congress on June 13, 2026, in Stockholm, Sweden.