Mitapivat-matching placebo

Phase 3

Mitapivat-matching placebo is one of 2 therapeutic programs targeting Pediatric Pyruvate Kinase Deficiency. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[2][1]

Indication

Pediatric Pyruvate Kinase DeficiencyActive, not recruiting

Pediatric Pyruvate Kinase DeficiencyPhase 3

Active, not recruiting

Competition

Competitive Landscape

1 competitor in Pediatric Pyruvate Kinase Deficiency

#DrugCompanyPhaseTrials

Aqvesme

AAgios Pharmaceuticals, Inc.

Phase 3

2 2

Mitapivat-matching placebo

AAgios Pharmaceuticals, Inc.

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q2 2029

Phase 3Active, not recruiting

Pediatric Pyruvate Kinase Deficiency

NCT05144256

Q1 2030

Phase 3Active, not recruiting

Pediatric Pyruvate Kinase Deficiency

NCT05175105

Clinical

Trials by Indication[1]

Pediatric Pyruvate Kinase Deficiency2 trials

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT05144256·49 enrolled

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 3·NCT05175105·30 enrolled

Sources

References

[1]
ClinicalTrials.gov — 2 trial records for Mitapivat-matching placebo. U.S. National Library of Medicine, National Institutes of Health.
[2]
Open Targets Platform — Drug–disease associations for Mitapivat-matching placebo. EMBL-EBI & Wellcome Sanger Institute.
[3]
SEC EDGAR — Agios Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Highest Phase: Phase 3[1]
Company: AAgios Pharmaceuticals, Inc.

Mitapivat-matching placebo

Status by Indication[2][1]

Competitive Landscape

Expected Milestones

Q2 2029

Q1 2030

Trials by Indication[1]

Pediatric Pyruvate Kinase Deficiency2 trials

References

Drug Info

Quick Facts

Related News