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# Review Extended on Agios sNDA for Sickle Cell Drug
- URL: https://www.fdaweb.com/review-extended-on-agios-snda-for-sickle-cell-drug/
- Published: 2026-10-30T20:42:01.000Z
- Updated: 2026-10-30T20:42:01.000Z
- Author: David McFarland
- Tags: Drugs

FDA has extended by three months its review of an Agios Pharmaceuticals supplemental NDA for mitapivat, an oral pyruvate kinase (PK) activator, for treating sickle cell disease. The user fee review action target date is now 2/1/2027.

The company says FDA recently requested additional information to evaluate whether a Risk Evaluation and Mitigation Strategy (REMS) is necessary for the drug. A company response, which included approximately nine months of updated safety data through 10/9, was submitted from the ongoing open-label extension periods of the RISE UP Phase 2 and Phase 3 trials.

“With this additional follow-up, there continue to be no cases of hepatocellular injury (HCI) among patients with sickle cell disease treated with mitapivat that show a pattern and severity similar to those observed in the clinical trials for mitapivat in adults with thalassemia,” the company says. “The benefit-risk profile of mitapivat in sickle cell disease remains unchanged.”

Subsequently, FDA informed Agios that the company’s response had been classified as a major amendment, resulting in a three-month review extension.

Agios says it will continue its labeling discussions with FDA, including elements of a potential REMS.

Currently, mitapivat (Pyrukynd)is approved for treating hemolytic anemia in adults with pyruvate kinase deficiency.