1. AGIO's sNDA for mitapivat remains under FDA review as of December 7, 2025. 2. FDA has not requested new efficacy or safety data from AGIO. 3. No timeline is provided by the FDA for the regulatory decision. 4. AGIO collaborates with FDA for labeling and REMS materials finalization.
FAQ
Why Neutral?
The uncertainty surrounding the FDA's decision may pause further price movements. Historical cases show regulatory delays can lead to stock stagnation.
How important is it?
AGIO’s stock is heavily influenced by FDA decisions; thus, the article has significant bearing.
Why Short Term?
The ongoing review and uncertainty will likely impact AGIO in the near future. Regulatory decisions can quickly alter investor sentiment and stock prices.
Agios Pharmaceuticals Updates on sNDA for Mitapivat in Thalassemia
CAMBRIDGE, Mass., Dec. 8, 2025 — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO) has announced that the U.S. Food and Drug Administration (FDA) has yet to make a regulatory decision regarding its supplemental New Drug Application (sNDA) for mitapivat. This application is intended for the treatment of adult patients suffering from both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The sNDA is currently under active review, following the Prescription Drug User Fee Act (PDUFA) goal date of December 7, 2025.
Regulatory Review Status
Agios is in close collaboration with the FDA to finalize essential labeling documents and the Risk Evaluation and Mitigation Strategy (REMS) materials for mitapivat. Importantly, the FDA has not requested any new or additional efficacy or safety data from Agios, nor has the company submitted such data.
As of now, the FDA has not provided a specific timeline for its regulatory decision, but Agios is committed to working diligently to conclude the review process of the sNDA.
About Agios Pharmaceuticals
Agios Pharmaceuticals is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. The company focuses on developing innovative therapies for patients with rare diseases. Their commitment is reflected in their vision to redefine the future of rare disease treatment by building partnerships with communities and creating transformative medicines.
- Company: Agios Pharmaceuticals, Inc.
- Stock Symbol: AGIO
- Headquarters: Cambridge, Massachusetts
Forward-Looking Statements
This announcement contains forward-looking statements as defined by The Private Securities Litigation Reform Act of 1995. These statements concern Agios' future expectations regarding its products, including the mitapivat regulatory process, and their strategic plans.
The following key phrases typically signify forward-looking statements:
- “Anticipate”
- “Expect”
- “Hope”
- “Goal”
- “Potential”
- “Strategy”
Investors should understand that several factors may cause actual results to differ significantly from those anticipated, including regulatory timing and patient enrollment in clinical trials. Such risks are further detailed in Agios' public filings with the Securities and Exchange Commission.
Contact Information
For Investor Inquiries:
Morgan Sanford
Vice President, Investor Relations
Agios Pharmaceuticals
Email: morgan.sanford@agios.com
For Media Inquiries:
Eamonn Nolan
Senior Director, Corporate Communications
Agios Pharmaceuticals
Email: eamonn.nolan@agios.com