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 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
A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)
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)
Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008
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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