The company took a major step toward an accelerated approval of mitapivat in sickle cell disease today. Agios needs to complete a confirmatory trial before a full traditional approval can be granted.

Agios stock skyrocketed closer to a profit-taking zone Tuesday after the FDA assigned its sickle cell drug a speedy review.

FDA's PDUFA goal date is November 1, 2026 If approved, mitapivat is positioned to become the first oral PK activator for patients with sickle cell disease CAMBRIDGE, Mass., July 07, 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 that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease with a Priority Review.

CAMBRIDGE, Mass., June 29, 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 company will host a conference call and live webcast on Thursday, July 30, 2026, at 8:00 a.m.

Agios' shares rose 35% in six months as Aqvesme gains traction, mitapivat expands globally and new ITP rights add to its rare blood disease push.

Agios Pharmaceuticals NASDAQ: AGIO used an investor webcast tied to the 2026 European Hematology Association Congress in Stockholm to highlight new clinical data for mitapivat across thalassemia and sickle cell disease, while outlining a broader rare disease strategy that now includes multiple hematology and metabolic programs.

Agios Pharmaceuticals, Inc. (AGIO) Discusses New Data and Strategic Expansion in Rare Hematology at EHA Investor Event Transcript

Mitapivat demonstrated statistically significant improvement in hemoglobin response compared with placebo, with rapid onset and durable effects New analyses showed patients in mitapivat arm had clinically meaningful reduction in transfusion burden compared with placebo Patients in mitapivat arm who achieved hemoglobin response had clinically meaningful benefits across measures of sickle cell pain crises, fatigue, and other patient-reported outcomes Mitapivat was well-tolerated, with a safety profile consistent with previous trials of mitapivat in sickle cell disease Company to host investor conference call and webcast today at 9:00 a.m. ET (3:00 p.m.

Agios gains global rights to cevidoplenib for ITP, adding a late-stage rare hematology asset with up to $1B U.S. peak sales potential.

Agios Pharmaceuticals, Inc. (AGIO) Discusses Global License Agreement for Cevidoplenib and Strategic Expansion in Rare Hematology Transcript