Trial started· ClinicalTrials.gov
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Mitapivat History and Updates
66 events · 2019 – present
Synced daily from openFDA and ClinicalTrials.gov. Last sync: .
3
FDA Approvals
0
Label Revisions
63
Trial Milestones
0
Pivotal Publications
Source: openFDA, ClinicalTrials.gov via AACT, PubMed · Hipa.ai, 2026-07-08Download 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
Trial started· ClinicalTrials.gov
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- o
Trial started· ClinicalTrials.gov
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Depend
Primary completion· ClinicalTrials.gov
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
Primary completion· ClinicalTrials.gov
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An E
Primary completion· ClinicalTrials.gov
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Depend
Primary completion· ClinicalTrials.gov
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- o
Primary completion· ClinicalTrials.gov
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Primary completion· ClinicalTrials.gov
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Past events
2026
Primary completion· ClinicalTrials.gov
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
Trial registered· ClinicalTrials.gov
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Trial registered· ClinicalTrials.gov
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Depend
Trial registered· ClinicalTrials.gov
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- o
Trial started· ClinicalTrials.gov
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
2025
FDA approval (supplemental)· FDA
Sponsor: AGIOS PHARMS INC. Class: Efficacy.
Results posted· ClinicalTrials.gov
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
Primary completion· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Primary completion· ClinicalTrials.gov
Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
Trial registered· ClinicalTrials.gov
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
Trial started· ClinicalTrials.gov
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
Results posted· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Results posted· ClinicalTrials.gov
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)· FDA
Sponsor: AGIOS PHARMS INC. Class: Labeling.
2024
Primary completion· ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regular
Primary completion· ClinicalTrials.gov
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
Trial registered· ClinicalTrials.gov
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
Trial started· ClinicalTrials.gov
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
Primary completion· ClinicalTrials.gov
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
Primary completion· ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly T
Primary completion· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Trial registered· ClinicalTrials.gov
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
2023
Trial started· ClinicalTrials.gov
Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
Primary completion· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N
Primary completion· ClinicalTrials.gov
A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With
Trial registered· ClinicalTrials.gov
Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
Trial started· ClinicalTrials.gov
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Trial registered· ClinicalTrials.gov
A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Trial started· ClinicalTrials.gov
A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With
2022
Trial registered· ClinicalTrials.gov
A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With
Trial started· ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly T
Trial started· ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regular
FDA approval· FDA
Sponsor: AGIOS PHARMS INC. Class: Type 1 - New Molecular Entity.
Trial started· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Trial registered· ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regular
2021
Trial started· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N
Trial registered· ClinicalTrials.gov
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly T
Trial started· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Trial registered· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Primary completion· ClinicalTrials.gov
A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Hea
Trial registered· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Trial registered· ClinicalTrials.gov
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-N
Trial started· ClinicalTrials.gov
A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Hea
Trial registered· ClinicalTrials.gov
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 started· ClinicalTrials.gov
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An E
Primary completion· ClinicalTrials.gov
A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants
Primary completion· ClinicalTrials.gov
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants
Trial registered· ClinicalTrials.gov
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An E
Trial registered· ClinicalTrials.gov
A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants
Trial started· ClinicalTrials.gov
A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants
Trial registered· ClinicalTrials.gov
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants
Trial started· ClinicalTrials.gov
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants
2019
Primary completion· ClinicalTrials.gov
Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348
Trial started· ClinicalTrials.gov
Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348
Trial registered· ClinicalTrials.gov
Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348
Trial started· ClinicalTrials.gov
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
Trial registered· ClinicalTrials.gov
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.