Ziftomenib
Medication
From Wikipedia, the free encyclopedia
Ziftomenib, sold under the brand name Komzifti, is an anti-cancer medication used for the treatment of acute myeloid leukemia.[1] Ziftomenib is a menin inhibitor.[1] It is taken by mouth.[1]
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| Trade names | Komzifti |
| Other names | KO-539; KO539 |
| AHFS/Drugs.com | komzifti |
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| Routes of administration | By mouth |
| Drug class | Antineoplastic |
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| Formula | C33H42F3N9O2S2 |
| Molar mass | 717.88 g·mol−1 |
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Ziftomenib blocks the interaction between two proteins, menin (MEN1) and KMT2A (also known as mixed lineage leukemia protein, MLL).[2][3]
Ziftomenib was approved for medical use in the United States in November 2025.[4][5]
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.[1]
Adverse effects
The US prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation, and embryo-fetal toxicity.[4]
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.[4] 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.[4]
The US Food and Drug Administration granted the application for ziftomenib priority review, breakthrough therapy, and orphan drug designations.[4]
Society and culture
Legal status
Ziftomenib was approved for medical use in the United States in November 2025.[6]
Names
Ziftomenib is the international nonproprietary name.[7][8]
Ziftomenib is sold under the brand name Komzifti.[6]
