The FDA has approved ziftomenib (Komzifti) for adults with relapsed or refractory acute myeloid leukemia (AML) harboring NPM1 mutations and lacking other effective treatments. This provides a new option for a challenging AML subgroup.
Approval was based on the phase 1/2 KOMET-001 trial, which showed a 21.4% complete remission (CR/CRh) rate, with responses lasting a median of five months. Among patients dependent on transfusions at baseline, 21.2% became independent from both red blood cell and platelet transfusions.
Ziftomenib was generally manageable, though nearly all patients experienced side effects, with 93% reporting grade 3 or higher events. Common adverse effects included diarrhea, febrile neutropenia, edema, nausea, differentiation syndrome, hypokalemia, anemia, pneumonia, and thrombocytopenia. Only 3% discontinued therapy due to toxicity.
This approval establishes ziftomenib as a targeted precision therapy for NPM1-mutated AML, offering hope for patients with limited treatment options.