The U.S. FDA granted approval to Kura Oncology and Kyowa Kirin’s ziftomenib (Komzifti) for relapsed or refractory acute myeloid leukemia with NPM1 mutations, clearing the drug ahead of its PDUFA date. Approval was supported by single‑arm Phase II Komet‑001 data showing complete remission plus partial hematologic recovery rates that exceeded historical benchmarks. Komzifti is the first menin inhibitor approved in this molecular subset and carries breakthrough designation; investigators reported a 22% CR/CRh rate and deep MRD negativity in responders. Safety signals included expected myelosuppression and differentiation syndrome, which were manageable under protocolized care. The approval establishes a targeted oral option for a genetically defined AML population and sets a precedent for regulatory reliance on single‑arm data in high‑unmet‑need hematologic malignancies.