Ziftomenib is an experimental drug for the treatment of cancer. It is being studied for use in patients with relapsed or refractory

acute myeloid leukaemia Acute myeloid leukemia (AML) is a cancer of the myeloid line of blood cells, characterized by the rapid growth of abnormal cells that build up in the bone marrow and blood and interfere with normal blood cell production. Symptoms may inclu ...

. Ziftomenib blocks the interaction between two proteins, menin (MEN1) and

KMT2A Histone-lysine ''N''-methyltransferase 2A, also known as acute lymphoblastic leukemia 1 (ALL-1), myeloid/lymphoid or mixed-lineage leukemia 1 (MLL1), or zinc finger protein HRX (HRX), is an enzyme that in humans is encoded by the ''KMT2A'' gene. ...

(also known as mixed lineage leukemia protein, MLL). This results in an inhibition of the proliferation of leukemic cells. Ziftomenib has been granted

breakthrough therapy designation Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "b ...

by the

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

References

{{antineoplastic-drug-stub Antineoplastic and immunomodulating drugs Trifluoroethyl compounds Sulfonamides Indoles Nitriles Thienopyrimidines Piperidines Piperazines Secondary amines