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Placebo Matching Mitapivat
Mitapivat activates pyruvate kinase R (PKR), an enzyme that increases ATP production in red blood cells to improve their survival and function.
Mitapivat activates pyruvate kinase R (PKR), an enzyme that increases ATP production in red blood cells to improve their survival and function. Used for Pyruvate kinase deficiency, Sickle cell disease.
At a glance
| Generic name | Placebo Matching Mitapivat |
|---|---|
| Sponsor | Agios Pharmaceuticals, Inc. |
| Drug class | Pyruvate kinase activator |
| Target | Pyruvate kinase R (PKR) |
| Modality | Small molecule |
| Therapeutic area | Hematology |
| Phase | Phase 3 |
Mechanism of action
Mitapivat is a small-molecule activator of pyruvate kinase R, the predominant form of pyruvate kinase in red blood cells. By enhancing PKR activity, the drug increases glycolytic ATP production, which strengthens the red blood cell membrane and reduces hemolysis. This mechanism is particularly beneficial in hemolytic anemias where red blood cell survival is compromised.
Approved indications
- Pyruvate kinase deficiency
- Sickle cell disease
Common side effects
- Headache
- Nausea
- Diarrhea
- Abdominal pain
Key clinical trials
- A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) (PHASE2, PHASE3)
- 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 (PHASE3)
- A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) (PHASE3)
- A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) (PHASE3)
- 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 (PHASE3)
- A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) (PHASE3)
- A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult Participants (PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Placebo Matching Mitapivat CI brief — competitive landscape report
- Placebo Matching Mitapivat updates RSS · CI watch RSS
- Agios Pharmaceuticals, Inc. portfolio CI