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6d ago

FDA Approves Komzifti, First Menin Inhibitor for Relapsed NPM1‑Mutant AML

Backed by early single-arm data showing modest remissions, the first menin inhibitor for NPM1‑mutant AML enters care with prominent safety warnings.

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FDA Approves Ziftomenib for NPM1+ R/R Acute Myeloid Leukemia
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Kura Oncology and Kyowa Kirin's Komzifti has gained FDA approval in adult patients with relapsed or refractory acute myeloid leukaemia (r/r AML) with a NPM1 mutation, joining Syndax's Revuforj as the second menin inhibitor on the AML market. Image credit: Ekahardiwito via Shutterstock.com.

Overview

  • The label covers adults with relapsed or refractory acute myeloid leukemia harboring a susceptible NPM1 mutation who lack satisfactory alternative options.
  • Approval rests on phase 1/2 KOMET‑001 results in 112 patients, with a 21.4% complete or near-complete remission rate and a median remission duration of 5 months at 4.2 months’ median follow‑up.
  • The prescribing information highlights risks including differentiation syndrome, QTc prolongation and embryo‑fetal toxicity, with two differentiation‑syndrome deaths reported in the trial.
  • Recommended dosing is 600 mg taken orally once daily until disease progression or unacceptable toxicity.
  • Additional signals included transfusion independence in a subset of patients and high rates of grade 3 or higher adverse events reported in the phase 2 cohort.