The US FDA has approved ziftomenib capsules for the treatment of adult patients with relapsed or refractory acute myeloid leukemia harboring a susceptible nucleophosmin 1 (NPM1) mutation and lacking satisfactory alternative treatment options. Ziftomenib is a menin inhibitor that blocks the interaction between menin and lysine [K]-specific methyltransferase 2A (KMT2A), thereby disrupting the oncogenic activity of the mutant NPM1 protein. Clinical evidence In a clinical trial of 112 adult patients, ziftomenib achieved a composite complete remission rate (CR plus CR with partial hematologic recovery, CRh) of 21.4 percent, with a median duration of 5 months.
The median time to first response among patients who achieved CR or CRh was 2.7 months. Notably, among the 24 patients who achieved CR or CRh, 88% responded within 6 months of treatment initiation. Dosage and administration 600 mg…
