Agios Announces New PDUFA Goal Date of February 1, 2027, for Mitapivat Sickle Cell Disease sNDA; Company Is Launch-Ready and in Labeling Discussions with FDA
Key Highlights
- ➤PDUFA goal date moved to February 1, 2027, after FDA review extension
- ➤FDA classified Agios’ response as a major amendment to mitapivat’s sNDA
- ➤FDA requested additional information to assess whether a REMS is necessary
- ➤Agios submitted approximately nine months of updated safety data with no comparable HCI cases
- ➤Agios says it is launch-ready and continues FDA labeling discussions
Expert Statements
Brian Goff, Chief Executive Officer of Agios
“We have strong conviction in mitapivat’s potential to transform care for people living with sickle cell disease, grounded in the comprehensive data from the RISE UP clinical program and further supported by the additional data we submitted in response to the FDA’s recent request. The FDA granted this application Priority Review, underscoring the serious need in this disease, and that urgency has not changed.”
Brian Goff, Chief Executive Officer of Agios
“Sickle cell disease affects approximately 100,000 people in the U.S. and is the largest indication we have pursued for mitapivat, and we expect it to be a significant growth driver for Agios. We are launch-ready, building on our commercial infrastructure and the strong relationships we have established over several years with hematologists, sickle cell centers, and patients. We will continue to work expeditiously with the goal of bringing mitapivat to patients with sickle cell disease as soon as possible.”
* FDA recently requested additional information to evaluate whether a REMS is necessary for mitapivat in sickle cell disease * FDA classified Agios’ response to the information request as a major amendment to the mitapivat sNDA, resulting in a three-month review extension * Mitapivat sNDA was submitted under the FDA’s accelerated approval pathway and granted Priority Review, with an original PDUFA goal date of November 1, 2026 * Agios will continue its labeling discussions with the FDA as part of the ongoing sNDA review, including elements of a potential REMS CAMBRIDGE, Mass., Oct. 30, 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 extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease. The PDUFA goal date for this sNDA, which was submitted under the FDA’s accelerated approval pathway and granted Priority Review, is now February 1, 2027.
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