Asset profile

mitapivat · Agios (AGIO)

Indication: anemia associated with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia
Application: sNDA sNDA
Also known as: PYRUKYND, AQVESME, tebapivat, PKR activator, AG-348

Company

Agios Pharmaceuticals Inc is a biopharmaceutical company focused on the field of cellular metabolism to create differentiated medicines for rare diseases, with a focus on classical hematology. The company's primary focus is to develop potentially transformativ…

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On the calendar

Jun 2026

Jun 13, 2026

95 days ago

AGIOAgios

Agios presents RISE UP Phase 3 mitapivat data at EHA 2026 plenary

Conference presentationOutcome not recordedCompany press release

Nov 2026

Nov 1, 2026

in 46 days

AGIOAgios

Agios: FDA sets Nov 1, 2026 PDUFA for mitapivat sNDA in sickle cell disease

Rescheduled — was Dec 7, 2025

FDA decision (PDUFA)ApprovedSEC 8-K

May 2027

~ May 2027

AGIOAgios

mitapivat: Phase 2 trial NCT06286046 expected to finish collecting primary-endpoint data (topline results usually follow)

Trial primary completionClinicalTrials.govMonth only

Feb 2028

Feb 28, 2028

in 530 days

AGIOAgios

mitapivat: Phase 1/Phase 2 trial NCT04610866 expected to finish collecting primary-endpoint data (topline results usually follow)

Trial primary completionClinicalTrials.gov

Mar 2029

~ Mar 2029

AGIOAgios

mitapivat: Phase 3 trial NCT07517133 expected to finish collecting primary-endpoint data (topline results usually follow)

Trial primary completionClinicalTrials.govMonth only

Jun 2029

~ Jun 2029

AGIOAgios

mitapivat: Phase 3 trial NCT07506863 expected to finish collecting primary-endpoint data (topline results usually follow)

Trial primary completionClinicalTrials.govMonth only

Aug 2029

~ Aug 2029

AGIOAgios

mitapivat: Phase 3 trial NCT07656415 expected to finish collecting primary-endpoint data (topline results usually follow)

Trial primary completionClinicalTrials.govMonth only

Timeline

Newest first.

Decision-grade

Jul 30, 2026

10:32 AM

AGIOAgios

Agios: FDA sets Nov 1, 2026 PDUFA for mitapivat sNDA in sickle cell disease

PDUFA date extendedSEC 8-K Open record

Notable

Jun 13, 2026

01:00 PM

AGIOAgios

Agios presents RISE UP Phase 3 mitapivat data at EHA 2026 plenary

Conference presentationCompany press release

Material

May 12, 2026

01:31 PM

AGIOAgios

Agios submits sNDA for mitapivat accelerated approval in sickle cell disease

Application submittedCompany press release Open record

Material

Apr 29, 2026

10:32 AM

AGIOAgios

Agios plans mitapivat sNDA submission for sickle cell disease in Q2 2026

Application submittedSEC 8-K Open record

Decision-grade

Feb 12, 2026

11:32 AM

AGIOAgios

Agios: FDA approves AQVESME (mitapivat) for thalassemia anemia

FDA approvalSEC 8-K Open record2 updates

Material

Jan 12, 2026

12:11 PM

AGIOAgios

Agios: pre-sNDA FDA meeting for mitapivat in sickle cell disease set for Q1 2026

PDUFA date setSEC 8-K Open record

Decision-grade

Oct 30, 2025

10:32 AM

AGIOAgios

Agios: FDA extends PYRUKYND thalassemia sNDA PDUFA to Dec 7, 2025

PDUFA date extendedSEC 8-K Open record

Readiness checklist

Public facts only. We never publish an approval probability.

Updated Sep 15, 2026

Primary endpoint metUnknown

Overall survival significant or trendingUnknown

US / Western patient shareMixed

106 of 396 sites in the United States

Advisory committee scheduledFavourable

No advisory committee announced

PDUFA date extendedConcern

Agios: FDA sets Nov 1, 2026 PDUFA for mitapivat sNDA in sickle cell disease

Manufacturing inspection recordUnknown

No public inspection record

Company language signals (label discussions)Unknown

Same-class precedentUnknown

Related companies

Each relationship is backed by the filing that stated it.

NewBridge Pharmaceuticals Not US-listedPartner

Avanzanite Bioscience B.V. Not US-listedPartner

AGIOAgiosOwner

Verify every date against the linked primary source. Informational only. Not investment advice. SimianX is not affiliated with the FDA or the SEC.

Registered trials

Registry ID	Phase	Status	Primary completion	Enrollment	Sites
NCT05175105	Phase 3	Active, not recruiting	Dec 13, 2024	30	11 of 19 sites in the United States
NCT04770753	Phase 3	Active, not recruiting	Nov 13, 2023	194	6 of 68 sites in the United States
NCT06286046	Phase 2	Withdrawn	May 1, 2027	0
NCT03853798	Phase 3	Completed	Jul 3, 2024	90	12 of 42 sites in the United States
NCT07656415	Phase 3	Recruiting	Aug 1, 2029	159	3 of 3 sites in the United States
NCT04770779	Phase 3	Active, not recruiting	Apr 11, 2024	258	10 of 75 sites in the United States
NCT05144256	Phase 3	Active, not recruiting	May 3, 2024	49	9 of 22 sites in the United States
NCT06924970	Phase 2	Active, not recruiting	May 12, 2026	59	10 of 20 sites in the United States
NCT07506863	Phase 3	Recruiting	Jun 1, 2029	54	3 of 3 sites in the United States
NCT07517133	Phase 3	Recruiting	Mar 1, 2029	45	4 of 4 sites in the United States
NCT04610866	Phase 1/2	Active, not recruiting	Feb 28, 2028	15	1 of 1 sites in the United States
NCT05935202	Phase 2	Not yet recruiting	Jul 15, 2025	25	0 of 2 sites in the United States
NCT05031780	Phase 2/3	Active, not recruiting	Oct 30, 2025	286	28 of 91 sites in the United States
NCT05490446	Phase 2	Active, not recruiting	Mar 17, 2026	87	9 of 45 sites in the United States
NCT07055243	Phase 2	Recruiting	Jun 30, 2026	9	0 of 1 sites in the United States