Mitapivat History and Updates

63 events · 2019 – present

Synced daily from openFDA and ClinicalTrials.gov. Last sync: June 18, 2026.

FDA Approvals

Label Revisions

Trial Milestones

Pivotal Publications

Source: openFDA, ClinicalTrials.gov via AACT, PubMed · Hipa.ai, 2026-06-02Download chart as PNG

This page tracks every FDA action, clinical trial milestone, and major publication for Mitapivat since 2019, drawn from openFDA, ClinicalTrials.gov, and PubMed.

All published updates (7)

2025

FDA approval (supplemental)Aqvesme (mitapivat) Gains FDA Supplemental Approval for Efficacy· Dec 23, 2025
Results postedMitapivat Pyruvate Kinase Deficiency Extension Study Posts Safety Results· Nov 18, 2025
Results postedMitapivat Shows Significant Transfusion Reduction in Thalassemia Trial· May 25, 2025

2024

Primary completionMitapivat Phase 3 Trial for Pediatric PKD Completes Primary Study· Dec 13, 2024
Primary completionMitapivat Phase 3 Trial for Pediatric PKD Completes Primary Study· May 3, 2024
Primary completionMitapivat Phase 3 for Transfusion-Dependent Thalassemia Reduces Transfusion…· Apr 11, 2024

2023

Primary completionMitapivat Phase 3 for Thalassemia Hits Primary Hemoglobin Response Endpoint· Nov 13, 2023

Upcoming & expected milestones

Primary completionJun 30, 2026· ClinicalTrials.gov

Phase 2 primary completion: NCT07055243

To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

Trial startedSep 30, 2026· ClinicalTrials.gov

Phase 3 trial started: NCT07506863

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- o

Trial startedSep 30, 2026· ClinicalTrials.gov

Phase 3 trial started: NCT07517133

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Depend

Primary completionMay 31, 2027· ClinicalTrials.gov

Phase 2 primary completion: NCT06286046

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

Primary completionFeb 28, 2028· ClinicalTrials.gov

Phase 1/Phase 2 primary completion: NCT04610866

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An E

Primary completionMar 31, 2029· ClinicalTrials.gov

Phase 3 primary completion: NCT07517133

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Depend

Primary completionJun 30, 2029· ClinicalTrials.gov

Phase 3 primary completion: NCT07506863

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- o

Primary completionAug 31, 2029· ClinicalTrials.gov

Phase 4 primary completion: NCT05777993

A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Past events

2026

Trial registeredApr 8, 2026· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT07517133

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Depend

Trial registeredApr 2, 2026· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT07506863

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- o

Trial startedMar 31, 2026· ClinicalTrials.gov

Phase 2 trial started: NCT06286046

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

2025

FDA approval (supplemental)Dec 23, 2025· FDA

FDA approved supplemental application for Aqvesme (mitapivat) — Efficacy (NDA 216196 S-3)

Sponsor: AGIOS PHARMS INC. Class: Efficacy.

Results postedNov 18, 2025· ClinicalTrials.gov

Trial results posted: NCT03853798

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Primary completionOct 30, 2025· ClinicalTrials.gov

Phase 2/Phase 3 primary completion: NCT05031780

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Primary completionJul 15, 2025· ClinicalTrials.gov

Phase 2 primary completion: NCT05935202

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

Trial registeredJul 8, 2025· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT07055243

To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

Trial startedJun 26, 2025· ClinicalTrials.gov

Phase 2 trial started: NCT07055243

To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.

Results postedMay 25, 2025· ClinicalTrials.gov

Trial results posted: NCT04770779

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Results postedJan 24, 2025· ClinicalTrials.gov

Trial results posted: NCT04770753

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N

FDA approval (supplemental)Jan 3, 2025· FDA

FDA approved supplemental application for Aqvesme (mitapivat) — Labeling (NDA 216196 S-4)

Sponsor: AGIOS PHARMS INC. Class: Labeling.

2024

Primary completionDec 13, 2024· ClinicalTrials.gov

Phase 3 primary completion: NCT05175105

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regular

Primary completionDec 3, 2024· ClinicalTrials.gov

Phase 1 primary completion: NCT06648824

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

Trial registeredOct 18, 2024· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT06648824

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

Trial startedOct 17, 2024· ClinicalTrials.gov

Phase 1 trial started: NCT06648824

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

Primary completionJul 3, 2024· ClinicalTrials.gov

Phase 3 primary completion: NCT03853798

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Primary completionMay 3, 2024· ClinicalTrials.gov

Phase 3 primary completion: NCT05144256

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly T

Primary completionApr 11, 2024· ClinicalTrials.gov

Phase 3 primary completion: NCT04770779

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Trial registeredFeb 29, 2024· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT06286046

A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy

2023

Trial startedDec 21, 2023· ClinicalTrials.gov

Phase 2 trial started: NCT05935202

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

Primary completionNov 13, 2023· ClinicalTrials.gov

Phase 3 primary completion: NCT04770753

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N

Primary completionJul 21, 2023· ClinicalTrials.gov

Phase 1 primary completion: NCT05610657

A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With

Trial registeredJul 7, 2023· ClinicalTrials.gov

Phase 2 trial registered on ClinicalTrials.gov: NCT05935202

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies

Trial startedJul 6, 2023· ClinicalTrials.gov

Phase 4 trial started: NCT05777993

A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Trial registeredMar 21, 2023· ClinicalTrials.gov

Phase 4 trial registered on ClinicalTrials.gov: NCT05777993

A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Trial startedJan 10, 2023· ClinicalTrials.gov

Phase 1 trial started: NCT05610657

A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With

2022

Trial registeredNov 9, 2022· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT05610657

A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With

Trial startedJun 8, 2022· ClinicalTrials.gov

Phase 3 trial started: NCT05144256

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly T

Trial startedJun 6, 2022· ClinicalTrials.gov

Phase 3 trial started: NCT05175105

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regular

FDA approvalFeb 17, 2022· FDA

FDA approved Aqvesme (mitapivat) (NDA 216196)

Sponsor: AGIOS PHARMS INC. Class: Type 1 - New Molecular Entity.

Trial startedFeb 11, 2022· ClinicalTrials.gov

Phase 2/Phase 3 trial started: NCT05031780

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Trial registeredJan 3, 2022· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT05175105

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regular

2021

Trial startedDec 20, 2021· ClinicalTrials.gov

Phase 3 trial started: NCT04770753

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N

Trial registeredDec 3, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT05144256

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly T

Trial startedNov 30, 2021· ClinicalTrials.gov

Phase 3 trial started: NCT04770779

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Trial registeredSep 2, 2021· ClinicalTrials.gov

Phase 2/Phase 3 trial registered on ClinicalTrials.gov: NCT05031780

A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)

Primary completionMar 19, 2021· ClinicalTrials.gov

Phase 1 primary completion: NCT04696393

A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Hea

Trial registeredFeb 25, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT04770779

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Trial registeredFeb 25, 2021· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT04770753

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N

Trial startedJan 6, 2021· ClinicalTrials.gov

Phase 1 trial started: NCT04696393

A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Hea

Trial registeredJan 6, 2021· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT04696393

A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Hea

2020

Trial startedDec 9, 2020· ClinicalTrials.gov

Phase 1/Phase 2 trial started: NCT04610866

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An E

Primary completionDec 3, 2020· ClinicalTrials.gov

Phase 1 primary completion: NCT04565678

A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants

Primary completionNov 23, 2020· ClinicalTrials.gov

Phase 1 primary completion: NCT04472832

A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants

Trial registeredNov 2, 2020· ClinicalTrials.gov

Phase 1/Phase 2 trial registered on ClinicalTrials.gov: NCT04610866

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An E

Trial registeredSep 25, 2020· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT04565678

A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants

Trial startedSep 21, 2020· ClinicalTrials.gov

Phase 1 trial started: NCT04565678

A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants

Trial registeredJul 15, 2020· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT04472832

A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants

Trial startedJun 17, 2020· ClinicalTrials.gov

Phase 1 trial started: NCT04472832

A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants

2019

Primary completionAug 14, 2019· ClinicalTrials.gov

Phase 1 primary completion: NCT03991312

Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348

Trial startedJun 20, 2019· ClinicalTrials.gov

Phase 1 trial started: NCT03991312

Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348

Trial registeredJun 19, 2019· ClinicalTrials.gov

Phase 1 trial registered on ClinicalTrials.gov: NCT03991312

Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348

Trial startedMar 21, 2019· ClinicalTrials.gov

Phase 3 trial started: NCT03853798

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Trial registeredFeb 26, 2019· ClinicalTrials.gov

Phase 3 trial registered on ClinicalTrials.gov: NCT03853798

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Sources

FDA approvals, label revisions, and recalls come from the public openFDA APIs (Drugs@FDA, Drug Label, Drug Enforcement). Drug-safety communications come from the FDA RSS feed. Trial milestones come from ClinicalTrials.gov via the AACT pg dump. Pivotal-publication events come from PubMed, filtered to high-impact journals linked to a Phase 3 trial in our inventory.