Chemistry:Ziftomenib

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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]

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

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]

References

  1. ↑ 1.0 1.1 1.2 1.3 Cite error: Invalid <ref> tag; no text was provided for refs named Komzifti FDA label
  2. ↑ "Ziftomenib". NCI Cancer Dictionary. National Cancer Institute. https://www.cancer.gov/publications/dictionaries/cancer-drug/def/ziftomenib. 
  3. ↑ "Menin inhibitor ziftomenib (KO-539) synergizes with drugs targeting chromatin regulation or apoptosis and sensitizes acute myeloid leukemia with MLL rearrangement or NPM1 mutation to venetoclax". Haematologica 108 (10): 2837–2843. October 2023. doi:10.3324/haematol.2022.282160. PMID 37102614. 
  4. ↑ 4.0 4.1 4.2 4.3 4.4 "FDA approves ziftomenib for relapsed or refractory acute myeloid leukemia with a NPM1 mutation". 13 November 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ziftomenib-relapsed-or-refractory-acute-myeloid-leukemia-npm1-mutation.  Public Domain This article incorporates text from this source, which is in the public domain.
  5. ↑ "Novel Drug Approvals for 2025". 13 November 2025. https://www.fda.gov/drugs/novel-drug-approvals-fda/novel-drug-approvals-2025. 
  6. ↑ 6.0 6.1 "Kura Oncology and Kyowa Kirin Announce FDA Approval of Komzifti (ziftomenib), the First and Only Once-Daily Targeted Therapy for Adults with Relapsed or Refractory NPM1-Mutated Acute Myeloid Leukemia" (Press release). Kura Oncology. 13 November 2025. Retrieved 14 November 2025 – via GlobeNewswire News Room.
  7. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information 36 (1). 2022. 
  8. ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 88". WHO Drug Information 36 (3). 2022. 

Further reading

  • "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. October 2024. doi:10.1016/S1470-2045(24)00386-3. PMID 39362248. 
  • Clinical trial number NCT04067336 for "First in Human Study of Ziftomenib in Relapsed or Refractory Acute Myeloid Leukemia" at ClinicalTrials.gov



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