NDAORALTABLETPriority Review
Approved
Feb 2022
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
16
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 18h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
PYRUKYND (mitapivat) is an oral pyruvate kinase activator approved in February 2022 for patients with pyruvate kinase deficiency, a rare genetic hemolytic anemia. It works by activating pyruvate kinase, a key glycolytic enzyme, to increase red blood cell energy and reduce hemolysis. This first-in-class mechanism addresses an unmet need in a limited but well-defined patient population.
$0.004BPart D
Peak15.1 years to LOEEarly-stage commercial performance in a rare disease segment with limited market penetration; small team size expected with focus on specialty pharmacy and hematology-focused sales.
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 (16)
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 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 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
Study of How Mitapivat Affects Midazolam Blood Levels in Healthy Participants
Started Oct 2024
20 enrolled
Healthy Participants
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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.