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Pyruvate Kinase Deficiency

5
Pipeline Programs
3
Companies
17
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
NCT04902833Completed18Est. Feb 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
Agios Pharmaceuticalsmitapivat
Agios PharmaceuticalsAG-348
Rocket PharmaceuticalsRP-L301
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios PharmaceuticalsAG-348
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Agios Pharmaceuticalsmitapivat
Rocket PharmaceuticalsRP-L301
Agios PharmaceuticalsBlood Draw
Agios PharmaceuticalsPyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Agios PharmaceuticalsPyruvate Kinase Deficiency Global Longitudinal Registry
CENTOGENEPyruvate Kinase Deficiency Epidemiological Study (PIECE)

Showing 15 of 17 trials with date data

Clinical Trials (17)

Total enrollment: 1,081 patients across 17 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 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

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

Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms

Start: Feb 2022Est. completion: Feb 202618 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
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
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 1,081 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.