Agios Pharmaceuticals confirmed that the U.S. Food and Drug Administration has not yet issued a decision on its supplemental New Drug Application (sNDA) for mitapivat in thalassemia, and that the FDA’s Prescription Drug User Fee Act (PDUFA) goal date of December 7, 2025 has passed without a verdict. The sNDA is supported by data from the Phase 3 ENERGIZE and ENERGIZE‑T trials and is the first step toward a U.S. launch of the oral pyruvate kinase activator in a rare blood disorder.
Agios explained that the delay stems from the FDA’s review of a major amendment—a proposed Risk Evaluation and Mitigation Strategy (REMS) addressing potential hepatocellular injury risks. The REMS submission, which prompted the September 2025 PDUFA extension, was considered a “major amendment” by the agency, and the agency has not yet completed its assessment of the REMS documentation. No new efficacy or safety data have been requested, and Agios is working closely with the FDA to finalize labeling and REMS details.
The postponement introduces uncertainty around the product’s launch timeline, which could defer revenue recognition and affect Agios’s cash‑runway calculations. The company reported a cash, cash equivalents, and marketable securities balance of $1.3 billion as of September 30, 2025, providing a runway that extends beyond 2026 even if the decision is delayed. However, the timing of the first sales of mitapivat in thalassemia is a key revenue driver, and any further delay could shift the company’s financial projections.
Investors reacted to the earlier September 2025 PDUFA extension with a 16% drop in Agios’s share price, reflecting concerns about the regulatory timeline. The current announcement is likely to reinforce caution among market participants, as the agency’s decision remains pending and the company’s launch plans are still uncertain.
Agios remains confident in the benefit‑risk profile of mitapivat and continues to collaborate with the FDA. The company expects a decision soon, but the exact timing remains unclear, leaving stakeholders to monitor the agency’s next communication for updates on the approval status.
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