New FDA Approvals: FDA Approves Pyrukynd

Feb. 17: The FDA approved Agios Pharmaceuticals, Inc.’s Pyrukynd (mitapivat) for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency. It is a first-in-class, oral PK activator and the first FDA-approved disease-modifying therapy for the disease. The agency gave the drug priority review and orphan drug designation. Dosing for the tablet is 5 mg twice daily. Its annual cost is $334,880.

Feb. 21: The FDA gave an additional approval to Foundation Medicine, Inc.’s FoundationOne CDx as a companion diagnostic to identify people with microsatellite instability high status solid tumors who may be candidates for treatment with Merck and Co., Inc.’s Keytruda (pembrolizumab). The company says it is the first FDA-approved diagnostic for this use. The CDx has 26 companion diagnostic claims and two group claims across 27 targeted therapies.