Aqvesme

(Mitapivat)

ApprovedSmall molecule

Aqvesme is one of 139 small molecule programs targeting Non-Transfusion-dependent Alpha-Thalassemia. It is currently in Approved.

Indications

Total Trials

Active Trials

138

Competitors

FDA Application

NDA/BLA/ANDA regulatory filings

Patents

Pipeline

Status by Indication[3][1]

Indication

Non-Transfusion-Dependent Alpha-ThalassemiaActive, not recruiting

Pediatric Pyruvate Kinase DeficiencyActive, not recruiting

Transfusion-Dependent Alpha-ThalassemiaActive, not recruiting

Pyruvate Kinase DeficiencyEnrolling by invitation

Safety and EfficacyNot yet recruiting

EfficacyRecruiting

Sickle Cell DiseaseRecruiting

Non-Transfusion-Dependent Alpha-ThalassemiaPhase 3

Active, not recruiting

Pediatric Pyruvate Kinase DeficiencyPhase 3

Active, not recruiting

Transfusion-Dependent Alpha-ThalassemiaPhase 3

Active, not recruiting

Pyruvate Kinase DeficiencyApproved

Enrolling by invitation

Safety and EfficacyPhase 2

Not yet recruiting

EfficacyPhase 2

Recruiting

Sickle Cell DiseasePhase 3

Recruiting

Competition

Competitive Landscape

149 competitors in Safety and Efficacy

#DrugCompanyPhaseTrials

Aqvesme

AAgios Pharmaceuticals, Inc.

Approved

1 2

Rituxan

Roche

Phase 3

8 3

Opdivo

Bristol-Myers Squibb

Phase 3

7 4

Humira

Novartis

Phase 3

3 5

Avastin

Roche

Phase 3

3 6

BEYFORTUS

Sanofi

Phase 3

1 7

Adriamycin

Pfizer

Phase 3

1 8

Oncovin

Eli Lilly

Phase 3

1 9

Neulasta

Amgen

Phase 3

1 10

Cytoxan

BBaxter Healthcare Corporation

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Not yet recruiting

Safety and Efficacy

NCT05935202

Q2 2027

Phase 2Recruiting

Efficacy

NCT07055243

Q1 2028

PreclinicalActive, not recruiting

Sickle Cell Disease

NCT04610866

Q4 2028

Phase 2Not yet recruiting

Sickle Cell Disease

NCT06286046

Phase 3Active, not recruiting

Non-Transfusion-Dependent Alpha-Thalassemia

NCT04770753

Clinical

Trials by Indication[1]

Sickle Cell Disease4 trials

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

Not yet recruiting·AAgios Pharmaceuticals, Inc.·Phase 2·NCT06286046·40 enrolled

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

Recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT07656415·159 enrolled

Evaluation of the Safety,Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

Active, not recruiting· Gilead logo

Gilead·Preclinical·NCT04610866·15 enrolled

Show 1 more trial

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

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Preclinical·NCT05031780·286 enrolled

Adult3 trials

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

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT03991312·28 enrolled

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

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT04472832·32 enrolled

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

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT04696393·26 enrolled

Pyruvate Kinase Deficiency2 trials

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

Completed·AAgios Pharmaceuticals, Inc.·Phase 3·NCT03853798·90 enrolled

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

Enrolling by invitation·AAgios Pharmaceuticals, Inc.·Approved·NCT05777993·59 enrolled

Pediatric Pyruvate Kinase Deficiency2 trials

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

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT05175105·30 enrolled

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

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT05144256·49 enrolled

Non-Transfusion-dependent Alpha-Thalassemia2 trials

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

Recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT07517133·45 enrolled

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)

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT04770753·194 enrolled

6 more indications

Transfusion-dependent Alpha-Thalassemia2 trials

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

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT04770779·258 enrolled

Recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT07506863·54 enrolled

Safety and Efficacy1 trial

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

Not yet recruiting·EEuroBloodNet Association·Phase 2·NCT05935202·25 enrolled

Moderate Hepatic Impairment1 trial

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

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT05610657·20 enrolled

ART1 trial

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

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT04565678·32 enrolled

Midazolam1 trial

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT06648824·20 enrolled

Efficacy1 trial

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

Recruiting·UUniversity Health Network, Toronto·Phase 2·NCT07055243·9 enrolled

Regulatory

FDA Approval Timeline[4]

Dec 23, 2025

AQVESME

NDA NDA216196

Sources

References

[1]
ClinicalTrials.gov — 20 trial records for Mitapivat. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Mitapivat compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Mitapivat. EMBL-EBI & Wellcome Sanger Institute.
[4]
OpenFDA — FDA approval records (NDA NDA216196). U.S. Food & Drug Administration.
[5]
FDA Orange Book — Patent and exclusivity data for Aqvesme. U.S. Food & Drug Administration.
[6]
SEC EDGAR — Agios Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Approved[1]
Company: AAgios Pharmaceuticals, Inc.

Patent Landscape[5]

Patent Cliff

Feb 24, 2031

Earliest patent expiration — generic entry possible after this date

Patent #Expires

RE49582Feb 24, 2031

9980961May 3, 2032

10632114May 3, 2032

9682080May 3, 2032

9193701Oct 26, 2032

4 more patents

11793806Apr 12, 2033

11234976Oct 11, 2038

11254652Nov 21, 2038

11878049Jul 31, 2041

Aqvesme

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q2 2027

Q1 2028

Q4 2028

Trials by Indication[1]

Sickle Cell Disease4 trials

Adult3 trials

Pyruvate Kinase Deficiency2 trials

Pediatric Pyruvate Kinase Deficiency2 trials

Non-Transfusion-dependent Alpha-Thalassemia2 trials

Transfusion-dependent Alpha-Thalassemia2 trials

Safety and Efficacy1 trial

Moderate Hepatic Impairment1 trial

ART1 trial

Midazolam1 trial

Efficacy1 trial

FDA Approval Timeline[4]

References

Drug Info

Quick Facts

Patent Landscape[5]

Related News