Pyruvate Kinase Deficiency
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: 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 [Phase 3]
An update on a Phase 3 pediatric trial evaluating the efficacy and safety of mitapivat in children with pyruvate kinase deficiency who are n…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: 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 [Phase 3]
Phase 3 clinical trial to evaluate the efficacy and safety of mitapivat in pediatric patients with pyruvate kinase deficiency, including a 5…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Pyruvate Kinase Deficiency Global Longitudinal Registry
A global longitudinal registry for Pyruvate Kinase Deficiency sponsored by Agios Pharmaceuticals, with the study currently active but not re…
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EMA approval · 2026-04-21EMA Authorised: Pyrukynd (mitapivat sulfate) — Agios Netherlands B.V.
The European Medicines Agency authorised Pyrukynd (mitapivat) for the treatment of pyruvate kinase deficiency—a genetic cause of hemolytic a…
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Clinical Trial to Evaluate the Efficacy of Gene Therapy for Pyruvate Kinase Deficiency [Phase 2]
A Phase 2 clinical trial evaluating a Rocket Pharmaceuticals gene therapy for Pyruvate Kinase Deficiency has been suspended.
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CLINICAL_TRIALS research · 2026-03-30Clinical Trial Update: A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study [Phase 4]
This document outlines a Phase 4 study providing continued access to mitapivat for participants who previously completed an Agios-sponsored …
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CLINICAL_TRIALS research · 2026-03-25Clinical Trial Update: Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms
This clinical trial update discusses a study of acquired pyruvate kinase deficiency in clonal myeloid neoplasms. The trial is currently acti…
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FDA approval · 2025-12-23FDA Approved: AQVESME — Supplemental Application (Agios Pharmaceuticals, Inc.)
The FDA approved a supplemental application for AQVESME (mitapivat) submitted by Agios Pharmaceuticals, Inc.
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CLINICAL_TRIALS trial_result · 2025-11-18Clinical Trial Update: Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 [Phase 3]
This update notes the completion of the Phase 3 extension study of AG-348 in adults with pyruvate kinase deficiency, with results posted.
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CLINICAL_TRIALS trial_result · 2025-09-05Clinical Trial Update: Gene Therapy for Pyruvate Kinase Deficiency (PKD) [Phase 1]
This document provides an update on a Phase 1 gene therapy trial for Pyruvate Kinase Deficiency, noting that the Phase 1 study has completed…
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FDA label_change · 2025-01-03FDA Approved: AQVESME — Supplemental Application (Agios Pharmaceuticals, Inc.)
The FDA approved a supplemental application for AQVESME (mitapivat) submitted by Agios Pharmaceuticals, resulting in changes to the product …
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CLINICAL_TRIALS trial_result · 2022-05-24Clinical Trial Update: A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) [Phase 3]
Phase 3 trial evaluating AG-348 (mitapivat) for efficacy and safety in not regularly transfused adults with pyruvate kinase deficiency has c…
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FDA approval · 2022-02-17FDA Approved: AQVESME — Original Application (Agios Pharmaceuticals, Inc.)
FDA approved AQVESME (mitapivat) from Agios Pharmaceuticals as the original application for the treatment of pyruvate kinase deficiency.
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CLINICAL_TRIALS trial_result · 2021-12-10Clinical Trial Update: A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) [Phase 3]
Phase 3 trial evaluating AG-348 in regularly transfused adults with pyruvate kinase deficiency has completed recruitment with results posted…
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CLINICAL_TRIALS trial_result · 2020-06-11Clinical Trial Update: A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency [Phase 2]
Phase 2 study of AG-348 (mitapivat) in adults with pyruvate kinase deficiency has completed and posted results.