# PYRUKYND (mitapivat) PDUFA Event: Sickle cell disease

Canonical URL: https://biopharmsignal.com/pdufa-calendar/AGIO/mitapivat-sickle-cell-disease

Ticker: AGIO

Company: Agios Pharmaceuticals, Inc.

Event date: 2026-11-01

Event type: FDA Decision

Stage: PDUFA

Result: Pending

Application type: sNDA

FDA decision expected. FDA assigned a November 1, 2026 PDUFA goal date to the mitapivat sNDA submitted under the accelerated approval pathway.

## Event Timeline

### November 1, 2026 — FDA 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](https://investor.agios.com/news-releases/news-release-details/us-fda-grants-priority-review-agios-snda-mitapivat-sickle-cell)

### July 7, 2026 — 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](https://investor.agios.com/news-releases/news-release-details/us-fda-grants-priority-review-agios-snda-mitapivat-sickle-cell)

### June 13, 2026 — 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](https://investor.agios.com/news-releases/news-release-details/agios-showcases-rise-phase-3-results-eha-2026-plenary-session)

### November 19, 2025 — 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](https://investor.agios.com/news-releases/news-release-details/agios-announces-topline-results-rise-phase-3-trial-mitapivat)

### February 11, 2022 — Phase 3 RISE UP study started

The pivotal RISE UP study began evaluating mitapivat in patients with sickle cell disease, including the hemolytic-anemia and vaso-occlusive-crisis endpoints supporting the sNDA.

[ClinicalTrials.gov — RISE UP study record (NCT05031780)](https://clinicaltrials.gov/study/NCT05031780)

## Regulatory Designations

- Priority Review FDA: [Agios — sNDA acceptance and Priority Review](https://investor.agios.com/news-releases/news-release-details/us-fda-grants-priority-review-agios-snda-mitapivat-sickle-cell)

- Orphan Drug EMA: [Agios — European Commission Orphan Medicinal Product designation](https://investor.agios.com/news-releases/news-release-details/european-commission-adopts-positive-decision-orphan-medicinal)

## Related Pages

- AGIO PDUFA calendar: https://biopharmsignal.com/pdufa-calendar/AGIO.md

- AGIO company profile: https://biopharmsignal.com/company/AGIO.md

- PDUFA calendar: https://biopharmsignal.com/pdufa-calendar.md

## Related News

- 2026-07-30 — Agios Reports Second Quarter 2026 Financial Results and Provides Business Update: https://biopharmsignal.com/news/AGIO/agios-reports-second-quarter-2026-financial-results-and-provides-business-update-2482094f.md

- 2026-07-21 — Agios Provides Update on Phase 2 Trial of Tebapivat in Sickle Cell Disease: https://biopharmsignal.com/news/AGIO/agios-provides-update-on-phase-2-trial-of-tebapivat-in-sickle-cell-disease-847f0155.md

- 2026-07-07 — U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease: https://biopharmsignal.com/news/AGIO/u-s-fda-grants-priority-review-to-agios-snda-for-mitapivat-in-sickle-cell-diseas-ca03247b.md

- 2026-06-29 — Agios to Host Second Quarter 2026 Financial Results Conference Call and Webcast on July 30 at 8:00 a.m. ET: https://biopharmsignal.com/news/AGIO/agios-to-host-second-quarter-2026-financial-results-conference-call-and-webcast--fc5d305e.md

- 2026-06-13 — Agios Showcases RISE UP Phase 3 Results at EHA 2026 Plenary Session Reinforcing Strong Anti-Hemolytic Profile of Mitapivat in Sickle Cell Disease: https://biopharmsignal.com/news/AGIO/agios-showcases-rise-up-phase-3-results-at-eha-2026-plenary-session-reinforcing--2ee47a9d.md