Pyrukynd FDA Approval History
Last updated by Judith Stewart, BPharm on Feb 17, 2022.
FDA Approved: Yes (First approved February 17, 2022)
Brand name: Pyrukynd
Generic name: mitapivat
Dosage form: Tablets
Company: Agios Pharmaceuticals, Inc.
Treatment for: Pyruvate Kinase Deficiency
Pyrukynd (mitapivat) is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
- Pyruvate kinase deficiency is a rare, inherited disease caused by mutations in the PKR genes, which cause deficiencies in red blood cell glycolysis. The condition presents as chronic hemolytic anemia.
- Pyrukynd works as an activator of both wild-type (normal) and mutated pyruvate kinase-R (PKR) enzymes involved in glycolysis.
- FDA approval of Pyruvate was based on the results from the ACTIVATE and ACTIVATE-T phase 3 studies. It is the first approved disease-modifying therapy for PK deficiency.
- Pyrukynd tablets are taken orally twice daily with or without food.
- Common adverse reactions including laboratory abnormalities in patients with PK deficiency include decreased estrone (males), increased urate, back pain, decreased estradiol (males), and arthralgia.
Development timeline for Pyrukynd
Further information
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