What Was Approved?
On November 13, 2025, the U.S. Food and Drug Administration approved Komzifti (ziftomenib) for adults with relapsed or refractory acute myeloid leukemia, or AML, with a susceptible NPM1 mutation who have no satisfactory alternative treatment options.
Komzifti is not approved for every patient with AML.
How Does Komzifti Work?
Komzifti is a menin inhibitor.
In certain NPM1-mutated AML cells, menin-dependent signaling helps support abnormal leukemia-cell growth. Ziftomenib targets this pathway.
How Is Komzifti Taken?
The recommended dose is:
600 mg by mouth once daily.
Treatment generally continues until disease progression or unacceptable toxicity.
What Did the Clinical Trial Show?
FDA approval was based on the KO-MEN-001 study involving 112 adults with relapsed or refractory NPM1-mutated AML.
The rate of complete remission or complete remission with partial hematologic recovery (CR/CRh) was 21.4%.
The median duration of response was about 5 months.
These results describe the study population and do not guarantee an individual response.
Does Komzifti Have a Boxed Warning?
Yes.
Komzifti carries a boxed warning for differentiation syndrome, a potentially life-threatening complication that can occur with certain leukemia treatments.
Possible symptoms may include:
- Fever
- Shortness of breath
- Rapid weight gain
- Swelling
- Low blood pressure
- Kidney problems
Prompt recognition and treatment are important.
What Other Safety Issues Are Important?
The FDA label also warns about:
- QTc prolongation
- Embryo-fetal toxicity
- Important drug interactions
Certain medicines that reduce stomach acid or prolong the QT interval may require avoidance or special management.
What Are Common Side Effects?
Common adverse reactions reported in the clinical trial included:
- Infections
- Bleeding
- Diarrhea
- Nausea
- Fatigue
- Edema
- Musculoskeletal pain
- Itching
- Febrile neutropenia
- Increased liver enzymes
Bottom Line
Komzifti (ziftomenib) is an FDA-approved oral menin inhibitor for certain adults with relapsed or refractory NPM1-mutated AML who do not have satisfactory alternative treatment options.
Because differentiation syndrome and other serious adverse effects can occur, treatment requires close oncology supervision and monitoring.
Sources
- U.S. Food and Drug Administration (FDA) — FDA Approves Ziftomenib for Relapsed or Refractory Acute Myeloid Leukemia With an NPM1 Mutation, November 13, 2025.
- U.S. Food and Drug Administration (FDA) — Drug Trials Snapshot: Komzifti.
- U.S. Food and Drug Administration (FDA) — Komzifti (ziftomenib) Prescribing Information, November 2025.