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Pyruvate Kinase Deficiency

5
Pipeline Programs
3
Companies
27
Clinical Trials
1 recruiting
2
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
1
0
1
3
Early DiscoveryClinical DevelopmentMarket

On Market (2)

Approved therapies currently available

Agios Pharmaceuticals
PYRUKYNDApproved
mitapivat
Agios Pharmaceuticals
Pyruvate Kinase Activator [EPC]oral2022
4M Part D
Agios Pharmaceuticals
AQVESMEApproved
mitapivat
Agios Pharmaceuticals
Pyruvate Kinase Activator [EPC]oral2022

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Agios Pharmaceuticals
Agios PharmaceuticalsMA - Cambridge
7 programs
1
1
1
PYRUKYND(mitapivat)Phase 45 trials
AG-348Phase 31 trial
AG-348Phase 21 trial
Blood DrawN/A1 trial
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)N/A1 trial
+2 more programs
Active Trials
NCT04902833Active Not Recruiting100Est. Dec 2026
NCT04964323Terminated15Est. Jun 2023
NCT04995315Completed13Est. Sep 2023
+9 more trials
Rocket Pharmaceuticals
2 programs
RP-L301PHASE_11 trial
RP-L301PHASE_21 trial
Active Trials
NCT04105166Completed4Est. Jun 2025
NCT06422351Suspended10Est. Jan 2029
CENTOGENE
CENTOGENEGermany - Rostock
1 program
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)N/A1 trial
Active Trials
NCT03866590Completed75Est. May 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios PharmaceuticalsAG-348
Agios Pharmaceuticalsmitapivat
Rocket PharmaceuticalsRP-L301
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios PharmaceuticalsAG-348
Agios Pharmaceuticalsmitapivat

Showing 15 of 27 trials with date data

Clinical Trials (27)

Total enrollment: 2,229 patients across 27 trials

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

Start: Jul 2023Est. completion: Aug 20296 patients
Phase 4Enrolling By Invitation

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

Start: Sep 2026Est. completion: Mar 203245 patients
Phase 3Not Yet Recruiting

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

Start: Sep 2026Est. completion: Jun 203254 patients
Phase 3Not Yet Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period

Start: Jun 2022Est. completion: Jun 202949 patients
Phase 3Active Not Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period

Start: Jun 2022Est. completion: Jan 203030 patients
Phase 3Active Not Recruiting

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

Start: Dec 2021Est. completion: Dec 2028194 patients
Phase 3Active Not Recruiting

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

Start: Nov 2021Est. completion: Jun 2029258 patients
Phase 3Active Not Recruiting

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

Start: Mar 2019Est. completion: Jul 202490 patients
Phase 3Completed

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Start: Jun 2018Est. completion: Nov 202027 patients
Phase 3Completed

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

Start: Feb 2022Est. completion: Feb 2030286 patients
Phase 2/3Active Not Recruiting

Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency

Start: Apr 2026Est. completion: Jan 202910 patients
Phase 2Suspended

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

Start: Mar 2026Est. completion: Dec 20280
Phase 2Withdrawn

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

Start: Jun 2025Est. completion: Jun 20279 patients
Phase 2Recruiting

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

Start: Jun 2015Est. completion: Apr 202552 patients
Phase 2Completed

Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants

Start: Oct 2024Est. completion: Dec 202420 patients
Phase 1Completed

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

Start: Jan 2023Est. completion: Jul 202320 patients
Phase 1Completed

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

Start: Jan 2021Est. completion: Mar 202126 patients
Phase 1Completed

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

Start: Sep 2020Est. completion: Dec 202032 patients
Phase 1Completed

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Start: Jul 2020Est. completion: Jun 20254 patients
Phase 1Completed

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

Start: Jun 2020Est. completion: Nov 202032 patients
Phase 1Completed

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

Start: Jun 2019Est. completion: Sep 201928 patients
Phase 1Completed

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Start: Feb 2022Est. completion: Dec 2026100 patients
N/AActive Not Recruiting
NCT04995315Agios PharmaceuticalsPyruvate Kinase Deficiency Global Longitudinal Registry

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

Start: Jul 2021Est. completion: Sep 202313 patients
N/ACompleted
NCT04964323Agios PharmaceuticalsPyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)

Start: Jul 2021Est. completion: Jun 202315 patients
N/ATerminated
NCT03866590CENTOGENEPyruvate Kinase Deficiency Epidemiological Study (PIECE)

Pyruvate Kinase Deficiency Epidemiological Study (PIECE)

Start: Jan 2020Est. completion: May 202175 patients
N/ACompleted
NCT03481738Agios PharmaceuticalsPyruvate Kinase Deficiency Global Longitudinal Registry

Pyruvate Kinase Deficiency Global Longitudinal Registry

Start: Apr 2018Est. completion: May 2027500 patients
N/AActive Not Recruiting
NCT02053480Agios PharmaceuticalsPyruvate Kinase Deficiency Natural History Study

Pyruvate Kinase Deficiency Natural History Study

Start: Dec 2013Est. completion: May 2020254 patients
N/ACompleted

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
1 actively recruiting trials targeting 2,229 patients
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.