Ziftomenib


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 and KMT2A.
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, 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.