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Ziftomenib

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Ziftomenib
Clinical data
Trade namesKomzifti
Other namesKO-539; KO539
AHFS/Drugs.comKomzifti
License data
Routes of
administration		By mouth
Drug classAntineoplastic
ATC code
  • None
Legal status
Legal status
Identifiers
  • 4-Methyl-5-[[4-[[2-(methylamino)-6-(2,2,2-trifluoroethyl)thieno[2,3-d]pyrimidin-4-yl]amino]piperidin-1-yl]methyl]-1-[(2S)-2-(4-methylsulfonylpiperazin-1-yl)propyl]indole-2-carbonitrile
CAS Number
PubChem CID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
PDB ligand
Chemical and physical data
FormulaC33H42F3N9O2S2
Molar mass717.88 g·mol−1
3D model (JSmol)
  • CC1=C(C=CC2=C1C=C(N2C[C@H](C)N3CCN(CC3)S(=O)(=O)C)C#N)CN4CCC(CC4)NC5=C6C=C(SC6=NC(=N5)NC)CC(F)(F)F
  • InChI=1S/C33H42F3N9O2S2/c1-21(43-11-13-44(14-12-43)49(4,46)47)19-45-25(18-37)15-27-22(2)23(5-6-29(27)45)20-42-9-7-24(8-10-42)39-30-28-16-26(17-33(34,35)36)48-31(28)41-32(38-3)40-30/h5-6,15-16,21,24H,7-14,17,19-20H2,1-4H3,(H2,38,39,40,41)/t21-/m0/s1
  • Key:BGGALFIXXQOTPY-NRFANRHFSA-N

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

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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

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The US prescribing information includes warnings and precautions for differentiation syndrome, QTc interval prolongation, and embryo-fetal toxicity.[4]

History

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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

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Ziftomenib was approved for medical use in the United States in November 2025.[6]

Names

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Ziftomenib is the international nonproprietary name.[7][8]

Ziftomenib is sold under the brand name Komzifti.[6]

References

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  1. ^ a b c d e "Komzifti Prescribing Information" (PDF). Kura Oncology. 2025.
  2. ^ "Ziftomenib". NCI Cancer Dictionary. National Cancer Institute.
  3. ^ Rausch J, Dzama MM, Dolgikh N, Stiller HL, Bohl SR, Lahrmann C, et al. (October 2023). "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. doi:10.3324/haematol.2022.282160. PMC 10543165. PMID 37102614.
  4. ^ a b c d e "FDA approves ziftomenib for relapsed or refractory acute myeloid leukemia with a NPM1 mutation". U.S. Food and Drug Administration (FDA). 13 November 2025. Retrieved 14 November 2025. Public Domain This article incorporates text from this source, which is in the public domain.
  5. ^ "Novel Drug Approvals for 2025". U.S. Food and Drug Administration (FDA). 13 November 2025. Retrieved 14 November 2025.
  6. ^ a b "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. ^ World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information. 36 (1). hdl:10665/352794.
  8. ^ World Health Organization (2022). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 88". WHO Drug Information. 36 (3). hdl:10665/363551.

Further reading

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  • 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.
[edit]
  • 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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