NPM1 mutations, found in about a third of adult acute myeloid leukaemias, misplace the nucleophosmin protein into the cytoplasm. They mean a better outlook without FLT3-ITD, a sensitive MRD marker, and since 2025 a targeted menin inhibitor.
Frameshift insertions in NPM1 exon 12 (type A, TCTG, in about 80 percent) are detected by PCR or sequencing and the transcript is tracked as measurable residual disease. Ziftomenib (Komzifti, 2025) is labelled for relapsed or refractory AML with a susceptible NPM1 mutation and no satisfactory alternative; revumenib (Revuforj) gained an NPM1-mutated relapsed or refractory indication in 2025 alongside its KMT2A-rearranged one. NPM1-mutated AML without FLT3-ITD is favourable-risk in ELN 2022 and is often spared transplant in first remission if MRD clears.
In plain words · NPM1 is the most common mutation in adult leukaemia. It moves a nuclear protein into the cytoplasm and, it turns out, makes the leukaemia dependent on menin.
An NPM1 mutation at diagnosis is, on its own, good news: these leukaemias respond well to chemotherapy, and the mutation can be tracked in your blood to catch relapse early. If the leukaemia does come back, ziftomenib or revumenib tablets are on label. Whether you also have a FLT3-ITD changes the plan, so both results are read together.
Written only from the label or guideline text cited on this page. Not medical advice; your own report and the reading your team gives it come first.
An NPM1 exon 12 frameshift insertion (type A, B, D or other) by PCR or sequencing of blood or marrow; quantitative PCR of the mutant transcript is used for residual disease.
“KOMZIFTI is indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 ( NPM1 ) mutation who have no satisfactory alternative treatment options”
Komzifti prescribing information| Threshold | Drug | Cancer | Regulator | Source |
|---|---|---|---|---|
| Susceptible NPM1 mutation, relapsed or refractory | Ziftomenib | NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia | FDA | label |
| NPM1 mutation, relapsed or refractory | Revumenib | NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia | FDA | label |
Matched on the name and aliases of the readout in the title, setting and summary of each trial; a match is a mention, not proof the readout was an entry criterion.
Shares NPM1 mutation, Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares FLT3-ITD (internal tandem duplication), Acute myeloid leukaemia and the tag biomarker.
Shares IDH1 R132 mutation, Acute myeloid leukaemia and the tag biomarker.
Shares NPM1 mutation, Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares Molecular response (MMR, MR4, treatment-free remission), Minimal / molecular residual disease (MRD) and the tag biomarker.
Shares NPM1 mutation, Ziftomenib, Revumenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia.
Shares IDH1 R132 mutation, Acute myeloid leukaemia and the tag biomarker.
Shares NPM1 mutation, Ziftomenib, NPM1-mutated and KMT2A-rearranged acute myeloid leukaemia, Acute myeloid leukaemia.