The U.S. Food and Drug Administration approved Kura Oncology and Kyowa Kirin’s oral menin inhibitor ziftomenib (Komzifti) for relapsed or refractory acute myeloid leukemia (AML) with NPM1 mutations. The approval, supported by single‑arm KOMET‑001 data, arrived ahead of the PDUFA date and marks Kura’s first marketed product. Komzifti produced clinically meaningful complete remission rates and measurable residual disease (MRD) negativity in a difficult‑to‑treat population, with a manageable safety profile and protocols to manage differentiation syndrome. Regulatory reviewers accepted single‑arm efficacy data given the high unmet need in r/r NPM1‑mutant AML. The decision creates immediate competition in a niche genomic subset and exemplifies regulatory flexibility for targeted oncology agents supported by deep biomarker data. For clinicians and trialists, Komzifti’s approval will alter treatment algorithms and provide a new targeted option for a genetically defined AML cohort.