Agios Pharmaceuticals (NASDAQ:AGIO) stock fell 20% after the U.S. Food and Drug Administration extended the review period for its thalassemia treatment by three months.
The FDA pushed back the Prescription Drug User Fee Act (PDUFA) target date for PYRUKYND (mitapivat) from September 7, 2025, to December 7, 2025, the company said. The delay follows Agios’ submission of a proposed Risk Evaluation and Mitigation Strategy (REMS) to address potential hepatocellular injury risk, which the FDA considered a major amendment to the supplemental New Drug Application (sNDA).
Agios stressed that the extension was not due to new safety or efficacy concerns. “We remain confident in the favorable benefit-risk profile of PYRUKYND in thalassemia,” said Brian Goff, Chief Executive Officer at Agios. “We look forward to continuing our collaborative engagement with the FDA, with the goal of bringing this disease-modifying oral medicine to adult patients with thalassemia in the U.S.”
PYRUKYND is an oral pyruvate kinase activator under evaluation for adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The application is supported by data from the global Phase 3 ENERGIZE and ENERGIZE-T trials.
The company is also aiming to expand PYRUKYND’s use, which is already approved for other rare blood disorders. The three-month delay pushes the potential launch for thalassemia patients into early 2026, assuming FDA approval.
Agios Pharmaceuticals stock price
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