Ziftomenib

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

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. ^ a b c d e "Komzifti- ziftomenib capsule". DailyMed. 9 December 2025. Retrieved 28 December 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. 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. ^ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 87". WHO Drug Information. 36 (1). 2022. hdl:10665/352794.
  8. ^ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 88". WHO Drug Information. 36 (3). 2022. hdl:10665/363551.

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.
  • 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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