Therapeutic Response Statement (Sensitivity)

This therapeutic response statement Supports the relationship that NPM1 p.W288Cfs*12 status confers therapeutic sensitivity to Ziftomenib in patients with Acute Myeloid Leukemia.

The U.S. Food and Drug Administration (FDA) granted approval to ziftomenib for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alternative treatment options. This approval is based on NCT04067336, an open-label, single-arm, multicenter clinical trial of 112 patients where eligibility criteria included NPM1 mutations, including Type A (c.860_863dupTCTG), B (c.863_864insCATG), and D (c.863_864insCCTG) mutations and other NPM1 mutations likely to result in cytoplasmic localization of the NPM1 protein.

This statement is based on a regulatory approval from the Food and Drug Administration:

KOMZIFTI is a menin inhibitor indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation who have no satisfactory alterantive treatment options.

Citation

Kura Oncology, Inc. Komzifti (ziftomenib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/220305s000lbl.pdf. Revised November 2025. Accessed December 23, 2025. Kura Oncology, Inc. Komzifti (ziftomenib) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/220305s000lbl.pdf. Revised November 2025. Accessed October 6, 2026.

Contributions

Date	Contributor	Description
2025-12-23	Van Allen lab	On 2025-11-13, the US FDA granted regular approval to ziftomenib for the treatment of patients with relapsed or refractory acute myeloid leukemia (AML) with susceptible NPM1 mutation who have no satisfactory alternative treatment options.
2025-11-13	Food and Drug Administration	Indication received traditional approval.