Regulatory approval

Published by the Food and Drug Administration.

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 is written in the approval document as:

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.

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Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Type Biomarker(s) Cancer type Therapy(ies)
Sensitivity (+) NPM1 p.W288Cfs*12 Acute Myeloid Leukemia Ziftomenib FDA