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mitapivat

Generic: MITAPIVAT SULFATE·Brand: AQVESME, Aqvesme, PYRUKYND, Pyrukynd

mitapivat: developed by 1 company · 15 clinical trials tracked.

mitapivat evidence translation

What we can confirm

What it means

Source-backed records connect mitapivat to AGIO; that link alone does not prove the drug works.

What to watch

Watch trial status, endpoints, and the next source-backed update.
BioSniper AIPage evidence connected

Prepared research question

How could mitapivat → AGIO affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence4 indications · 0 study sponsors

Related indications

Hemolytic anemia in adults with PK deficiency; being evaluated for thalassemia, sickle cell disease (SCD), and pediatric patients with PK deficiencyAnemiaSickle Cell DiseaseThalassemia

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (1)
CompanyPhase
AGIOS PHARMACEUTICALS, INC. (AGIO)
Hemolytic anemia in adults with PK deficiency; being evaluat…
Approved
Clinical Trials (15)showing 10 most recent

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Non-Transfusion-Dependent Thalassemia

NCT07517133·Phase 3·AGIO
Recruiting

Primary endpoint: Percentage of Participants Who Achieved a Hemoglobin (Hb) Response

InterventionalRandomizedquadruple45 targetStarted 2026-09Completion 2032-034 sites · United StatesAgios Pharmaceuticals, Inc.

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia

NCT07506863·Phase 3·AGIO
Recruiting

Primary endpoint: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48

InterventionalRandomizedquadruple54 targetStarted 2026-09Completion 2032-063 sites · United StatesAgios Pharmaceuticals, Inc.

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease

NCT07656415·Phase 3·AGIO
Recruiting

Primary endpoint: Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52

InterventionalRandomizeddouble159 targetStarted 2026-07Completion 2030-083 sites · United StatesAgios Pharmaceuticals, Inc.

A Phase 2, Open-label, Multicenter Study of Mitapivat in Subjects With Sickle Cell Disease and Nephropathy (RESIST)

NCT06286046·Phase 2·AGIO
Withdrawn

Primary endpoint: Percentage of Participants With Albumin Creatinine Ratio (ACR) Response

Interventionalopen0 targetStarted 2026-03Completion 2028-12Agios Pharmaceuticals, Inc.

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies - Canada (SATISFY-CA)

NCT07055243·Phase 2·AGIO
Recruiting

Primary endpoint: To evaluate safety of mitapivat

Interventionalopen9 targetStarted 2025-06Completion 2027-061 site · CanadaUniversity Health Network, Toronto

A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study

NCT05777993·Phase 4·AGIO
Enrolling by invitation

Primary endpoint: Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study

Interventionalopen59 targetStarted 2023-07Completion 2029-0823 sites · Belgium, Brazil, Canada, France…Agios Pharmaceuticals, Inc.

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period

NCT05144256·Phase 3·AGIO
Active, not recruiting

Primary endpoint: Percentage of Participants Achieving Transfusion Reduction Response (TRR)

InterventionalRandomizedquadruple49 enrolledStarted 2022-06Completion 2029-0622 sites · Canada, Czechia, Denmark, Germany…Agios Pharmaceuticals, Inc.

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

NCT05175105·Phase 3·AGIO
Active, not recruiting

Primary endpoint: Percentage of Participants Achieving a Hemoglobin (Hb) Response

InterventionalRandomizedquadruple30 enrolledStarted 2022-06Completion 2030-0119 sites · Canada, France, Germany, Netherlands…Agios Pharmaceuticals, Inc.

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

NCT05031780·Phase 2/Phase 3·AGIO
Active, not recruiting

Primary endpoint: Phase 2: Percentage of Participants With Hemoglobin (Hb) Response

InterventionalRandomizedquadruple286 targetStarted 2022-02Completion 2030-0250 sites · Belgium, Brazil, Canada, France…Agios Pharmaceuticals, Inc.

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)

NCT04770753·Phase 3·AGIO
Active, not recruiting

Primary endpoint: Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With Baseline

InterventionalRandomizedquadruple194 enrolledStarted 2021-12Completion 2028-1250 sites · Brazil, Bulgaria, Canada, Denmark…Agios Pharmaceuticals, Inc.

Frequently asked questions

Who is developing mitapivat?

mitapivat is being developed by AGIOS PHARMACEUTICALS, INC..

How many clinical trials involve mitapivat?

BioSniper tracks 15 clinical trials involving mitapivat.