Pyruvate Kinase Deficiency
Pipeline by Development Stage
On Market (2)
Approved therapies currently available
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
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
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
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)
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
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
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic Function
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
A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Participants
Gene Therapy for Pyruvate Kinase Deficiency (PKD)
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants
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
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Pyruvate Kinase Deficiency Epidemiological Study (PIECE)
Pyruvate Kinase Deficiency Global Longitudinal Registry
Pyruvate Kinase Deficiency Natural History Study
Phase Legend
Key Insights
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