Agios Provides Update on U.S. sNDA for Mitapivat in Thalassemia
Rhea-AI Summary
Agios (Nasdaq: AGIO) announced that the U.S. FDA has not issued a regulatory decision on the supplemental NDA for mitapivat in adult non‑transfusion‑dependent and transfusion‑dependent alpha‑ or beta‑thalassemia; the application remains under active review.
The FDA's PDUFA goal date was December 7, 2025. Agios said the agency has not requested, and the company has not submitted, any new efficacy or safety data, and that the companies are collaborating to finalize labeling and REMS materials. The FDA has not provided a timeline for its decision and Agios continues to work expeditiously to conclude the review.
Positive
- PDUFA goal date of December 7, 2025 established
- FDA has not requested additional efficacy or safety data
Negative
- FDA has not issued a regulatory decision as of December 8, 2025
- FDA provided no timeline for a final decision
Key Figures
Market Reality Check
Peers on Argus 1 Up
Within Biotechnology peers, moves were mixed: APGE up 2.5%, while names like DNLI, IRON, TVTX, and FOLD were down modestly. Momentum scanner activity was limited, with only OCUL flagged up 12.96% and no related news, suggesting this FDA timing update is stock-specific rather than part of a sector-wide move.
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Nov 19 | Phase 3 data | Positive | -50.9% | Topline RISE UP Phase 3 sickle cell data met primary hemoglobin endpoint. |
| Nov 03 | Conference preview | Positive | -7.2% | Announcement of new mitapivat data presentations at the 67th ASH Meeting. |
| Oct 30 | Earnings update | Positive | +2.9% | Q3 2025 results with PYRUKYND revenue growth and strong cash balance. |
| Oct 17 | Regulatory opinion | Positive | +1.9% | Avanzanite highlighted positive CHMP opinion for PYRUKYND in thalassemia. |
| Oct 17 | Regulatory opinion | Positive | +1.9% | Agios announced EMA CHMP positive opinion for PYRUKYND in thalassemia. |
Recent history shows sharp negative reactions to otherwise positive or mixed clinical data, especially for mitapivat in sickle cell disease, while earnings and regulatory milestones around PYRUKYND and thalassemia have seen modestly positive, more aligned price responses.
Over the last few months, Agios has focused on mitapivat across multiple indications and regions. On Oct 17, positive CHMP opinions for PYRUKYND in thalassemia and a European commercialization agreement with Avanzanite coincided with modest gains of 1.95%. The Oct 30 Q3 2025 report highlighted PYRUKYND net revenue of $12.9M and cash of $1.3B, with shares up 2.91%. In contrast, the Nov 19 RISE UP Phase 3 sickle cell data, which met its primary endpoint, saw a steep -50.89% move, underscoring market sensitivity to clinical details versus today’s regulatory timing update.
Market Pulse Summary
This announcement clarifies that the FDA has not yet issued a decision on the mitapivat sNDA in thalassemia, even though the original Dec 7, 2025 PDUFA date has passed. The filing remains under active review, with no new efficacy or safety data requested, and Agios working on labeling and REMS materials. Investors may track future FDA communications, progress on European decisions, and the company’s cash position of $1.3B when assessing regulatory and funding runway.
Key Terms
supplemental new drug application regulatory
pdufa regulatory
AI-generated analysis. Not financial advice.
CAMBRIDGE, Mass., Dec. 08, 2025 (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 not yet issued a regulatory decision on the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia and that the sNDA remains under active review. The Prescription Drug User Fee Act (PDUFA) goal date issued by the FDA was December 7, 2025.
Agios is collaborating closely with the FDA to finalize the labeling documents and Risk Evaluation and Mitigation Strategy (REMS) materials. The FDA has not requested, and the company has not submitted, any new or additional efficacy or safety data.
The FDA has not provided a timeline for its regulatory decision. Agios continues to work expeditiously with the FDA to conclude the review of the sNDA.
About Agios: Fueled by Connections to Transform Rare Diseases™
At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of Agios’ products, including mitapivat, its interactions with regulators, including the FDA, and its strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
Contacts:
Investor Contact
Morgan Sanford, Vice President, Investor Relations
Agios Pharmaceuticals
morgan.sanford@agios.com
Media Contact
Eamonn Nolan, Senior Director, Corporate Communications
Agios Pharmaceuticals
eamonn.nolan@agios.com
FAQ
What is the current FDA status of Agios's mitapivat sNDA for thalassemia (AGIO)?
What was the PDUFA goal date for Agios's mitapivat sNDA (AGIO)?
Did the FDA request additional efficacy or safety data for AGIO's mitapivat sNDA?
Has the FDA given a timeline for its decision on Agios's mitapivat sNDA (AGIO)?
What are Agios and the FDA currently working on for the mitapivat sNDA (AGIO)?
