NDAORALTABLETPriority Review
Approved
Feb 2022
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
17
Data completeness
5/8 — Partial
Priority Review
PYRUKYND (mitapivat) by Agios Pharmaceuticals is pyruvate kinase activators [moa]. First approved in 2022.
Drug data last refreshed 9h ago · AI intelligence enriched 3d ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PYRUKYND (mitapivat) is an oral pyruvate kinase activator approved in February 2022 for treating pyruvate kinase deficiency, a rare genetic hemolytic anemia. It works by activating pyruvate kinase enzyme activity to increase red blood cell lifespan and reduce hemolysis. The drug addresses an unmet need in a niche rare disease population with limited prior treatment options.
$0.004BPart D
Peak14.9 years to LOEEarly-stage commercial performance in a rare disease market with small patient population suggests focused, specialized sales and marketing teams rather than broad franchise infrastructure.
Mechanism of Action
Pyruvate Kinase Activators
Pharmacologic Class:
Pyruvate Kinase Activator
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (17)
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
Started Sep 2026
45 enrolled
Non-Transfusion-dependent Alpha-ThalassemiaNon-Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
Started Sep 2026
54 enrolled
Transfusion-dependent Alpha-ThalassemiaTransfusion-dependent Beta-Thalassemia
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
Started Jul 2026
159 enrolled
Sickle Cell Disease
A Study of Mitapivat in Participants With Sickle Cell Disease and Nephropathy
Started Mar 2026
0Sickle Cell DiseaseNephropathy
To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
Started Jun 2025
9 enrolled
Anemia
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.