Last reviewed · How we verify
AG-348
AG-348 activates pyruvate kinase R (PKR), an enzyme that increases ATP production in red blood cells to improve their survival and reduce hemolysis.
AG-348 activates pyruvate kinase R (PKR), an enzyme that increases ATP production in red blood cells to improve their survival and reduce hemolysis. Used for Pyruvate kinase deficiency, Sickle cell disease.
At a glance
| Generic name | AG-348 |
|---|---|
| Also known as | AG-348 will be administered as a single oral dose on Day 1., 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
AG-348 is a direct activator of pyruvate kinase R (PKR), the predominant form of pyruvate kinase in red blood cells. By enhancing PKR activity, the drug increases glycolytic ATP production in erythrocytes, which stabilizes 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 of Mitapivat in Participants With Sickle Cell Disease and Nephropathy (PHASE2)
- 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 to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study (PHASE4)
- A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia (PHASE2)
- 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)
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:
- AG-348 CI brief — competitive landscape report
- AG-348 updates RSS · CI watch RSS
- Agios Pharmaceuticals, Inc. portfolio CI