Mitapivat
Mitapivat is a pyruvate kinase activator used to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency. It works by allosterically binding to the pyruvate kinase tetramer, increasing PK activity to help reduce oxidative stress and improve red blood cell survival.
Mitapivat is a pyruvate kinase activator used to treat pyruvate kinase deficiency-related hemolytic anemia by increasing red blood cell pyruvate kinase activity.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- B06AX04
- RxNorm
- 2594468
- Generic
- Mitapivat
- Class
- atc-b06
- Brands
- Pyrukynd, Aqvesme
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- tablet
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Treatment of hemolytic anemia in adults with pyruvate kinase deficiency (PKD), a rare genetic disorder characterized by defective pyruvate kinase enzyme activity leading to premature red blood cell destruction.
Mitapivat is a small molecule that allosterically binds to the pyruvate kinase tetramer, activating the enzyme. This helps restore energy metabolism in red blood cells, reduces oxidative stress caused by globin chain imbalances during erythropoiesis, and improves erythrocyte survival.
This is an oral medication approved specifically for PK deficiency. Patients should have their hemoglobin and hemolysis markers monitored during treatment. Use with strong CYP3A inducers may decrease mitapivat exposure. Common side effects may include urinary tract infection, back pain, and fatigue.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy