PYRUKYND (mitapivat)
Sickle cell disease · Patients aged 16 years or older with sickle cell disease
AGIO · Agios Pharmaceuticals, Inc.
Regulatory designations
Full timeline
5 milestonesFDA decision expected
FDA assigned a November 1, 2026 PDUFA goal date to the mitapivat sNDA submitted under the accelerated approval pathway.
Agios — sNDA acceptance and PDUFA date↗FDA accepted the sNDA with Priority Review
FDA accepted the supplemental application based on the global RISE UP Phase 2 and Phase 3 studies in patients aged 16 years or older.
Agios — sNDA acceptance and Priority Review↗Detailed Phase 3 results were presented
At EHA 2026, Agios reported a statistically significant hemoglobin response and additional analyses showing lower transfusion burden with mitapivat.
Agios — RISE UP results at EHA 2026↗RISE UP Phase 3 topline results were reported
RISE UP met its hemoglobin-response primary endpoint and two key secondary endpoints, while the pain-crisis primary endpoint did not reach statistical significance.
Agios — RISE UP Phase 3 topline results↗Related News
Agios to Host Third Quarter 2026 Financial Results Conference Call and Webcast on November 5 at 8:00 a.m. ET
Jul 30, 2026Agios Reports Second Quarter 2026 Financial Results and Provides Business Update
Jul 21, 2026Agios Provides Update on Phase 2 Trial of Tebapivat in Sickle Cell Disease
Jul 7, 2026U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease
Jun 29, 2026Agios to Host Second Quarter 2026 Financial Results Conference Call and Webcast on July 30 at 8:00 a.m. ET