FDA Approves Ziftomenib for R/R NPM1-Mutated AML

Ziftomenib (Komzifti; Kura Oncology and Kyowa Kirin), an oral, once-daily, selective menin inhibitor, has received approval from the FDA for the treatment of adult patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) harboring a susceptible NPM1 mutation.^1,2

This marks the second menin inhibitor to be approved for this indication,³ providing a new targeted pathway option for a high-risk population with historically poor outcomes. The approval is based on positive data from the pivotal phase 2 KOMET-001 trial, which demonstrated clinically meaningful response rates and a favorable benefit-risk profile in a heavily pretreated patient cohort.

References

1. FDA approves ziftomenib for relapsed or refractory acute myeloid leukemia with a NPM1 mutation. FDA. November 13, 2025. Accessed November 13, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ziftomenib-relapsed-or-refractory-acute-myeloid-leukemia-npm1-mutation

2. Kura Oncology and Kyowa Kirin announce FDA approval of Komzifti (ziftomenib), the first and only once-daily targeted therapy for adults with relapsed or refractory NPM1-mutated acute myeloid leukemia. News release. Kura Oncology. November 13, 2025. Accessed November 13, 2025. https://ir.kuraoncology.com/news-releases/news-release-details/kura-oncology-and-kyowa-kirin-announce-fda-approval-komziftitm

3. Joszt L. Revumenib granted FDA approval for R/R NPM1-mutated AML. AJMC®. October 24, 2025. Accessed November 13, 2025. https://www.ajmc.com/view/revumenib-granted-fda-approval-for-r-r-npm1-mutated-aml