Ziftomenib, sold under the brand name Komzifti, is an anti-cancer medication used for the treatment of acute myeloid leukemia. Ziftomenib is a menin inhibitor. It is taken by mouth. Ziftomenib blocks the interaction between two proteins, menin (MEN1) and KMT2A (also known as mixed lineage leukemia protein, MLL). Ziftomenib was approved for medical use in the United States in November 2025.
Medical uses Ziftomenib is indicated for the treatment of adults with relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 mutation who have no satisfactory alternative treatment options.
Adverse effects The US prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation, and embryo-fetal toxicity.
History Efficacy was evaluated in KO-MEN-001 (NCT04067336), an open-label, single, arm, multi-center trial in 112 adults with relapsed or refractory acute myeloid leukemia with an nucleophosmin 1 mutation identified using next-generation sequencing or polymerase chain reaction. Participants with nucleophosmin 1 mutations, including type A, B, and D mutations and other nucleophosmin 1 mutations likely to result in cytoplasmic localization of the nucleophosmin 1 protein, were enrolled. The US Food and Drug Administration granted the application for ziftomenib priority review, breakthrough therapy, and orphan drug designations.
Society and culture
Legal status Ziftomenib was approved for medical use in the United States in November 2025.
Names Ziftomenib is the international nonproprietary name. Ziftomenib is sold under the brand name Komzifti.
References
Further reading Wang ES, Issa GC, Erba HP, Altman JK, Montesinos P, DeBotton S, et al. (October 2024). "Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial". The Lancet. Oncology. 25 (10): 1310–1324. doi:10.1016/S1470-2045(24)00386-3. PMID 39362248.
External links Clinical trial number NCT04067336 for "First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia" at ClinicalTrials.gov


