VivoSeq
Hematology

Pyruvate Kinase Deficiency

1

Pipeline Programs↗

3

Active Trials↗

2

Companies Involved↗

Pyruvate Kinase Deficiency is an early-stage landscape with 1 program across 2 companies. The most active players are Agios Pharmaceuticals, Inc., Rocket Pharmaceuticals. 1 therapy is approved.

Intelligence

Pipeline Metrics

Competitive Density

19/100

Pipeline Velocity

0/100

Mech. Concentration

N/A

Completion Rate

100/100

Attrition Rate

0/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

A

Agios Pharmaceuticals, Inc.

1 program

ApprovedRocket Pharmaceuticals logo

Rocket Pharmaceuticals

0 programs

Preclinical

Pipeline

Programs by Phase

Preclinical
No programs
Phase 1
No programs
Phase 2
No programs
Phase 3
No programs
Approved1

Mitapivat

Agios Pharmaceuticals, Inc.

Analysis

Trial Status

7

clinical trials

Completed4 · 57%
Not yet recruiting1 · 14%
Active, not recruiting1 · 14%
Enrolling by invitation1 · 14%

Clinical Trials

Trial Activity

7 trials

Pyruvate Kinase Deficiency Global Longitudinal Registry
Active, not recruiting·AAgios Pharmaceuticals, Inc.·N/A·NCT03481738·500 enrolled
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
Not yet recruiting·Rocket Pharmaceuticals logoRocket Pharmaceuticals·Phase 2·NCT06422351·10 enrolled
A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study
Enrolling by invitation·AAgios Pharmaceuticals, Inc.·Approved·NCT05777993·6 enrolled
A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Completed·AAgios Pharmaceuticals, Inc.·Phase 3·NCT03548220·80 enrolled
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
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
Completed·Rocket Pharmaceuticals logoRocket Pharmaceuticals·Phase 1·NCT04105166·4 enrolled
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
Completed·AAgios Pharmaceuticals, Inc.·Phase 3·NCT03559699·27 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)