New Targeted Drug Approved for Certain Adults With Relapsed or Refractory AML With an NPM1 Mutation

Komzifti improves outcomes by offering a targeted oral option that can produce deep, sometimes durable remissions and reduce transfusion needs in heavily pretreated adults with NPM1 mutated relapsed or refractory AML, a group that typically has poor responses to existing salvage therapies

Charles H. Weaver, MD
1–2 minutes
Home » Leukemia » Acute Leukemia » New Targeted Drug Approved for Certain Adults With Relapsed or Refractory AML With an NPM1 Mutation

The FDA has approved a new targeted treatment called ziftomenib (Komzifti) for adults with acute myeloid leukemia (AML) that has come back or not responded to prior therapy and has a specific change (mutation) in a gene called NPM1. This medicine is intended for people who no longer have good standard treatment options and works by blocking a protein called menin, which certain leukemia cells rely on to survive and grow.​

In the KO-MEN-001 clinical trial of 112 adults with relapsed or refractory NPM1‑mutated AML, about 1 in 5 patients achieved a complete remission or a near-complete remission with blood count recovery, and these responses lasted a median of 5 months. Some patients who needed regular red blood cell or platelet transfusions before starting treatment were able to become transfusion‑independent during therapy, meaning they no longer needed transfusions for a period of time.​

Ziftomenib also has important safety considerations. The prescribing information warns about the risk of differentiation syndrome (a potentially serious inflammatory reaction), changes in heart rhythm (QTc prolongation), and possible harm to an unborn baby, so anyone considering this treatment should review the full medication guide and discuss potential benefits and risks carefully with their leukemia team.​

About Ziftomenib

Komzifti (ziftomenib) is an oral targeted therapy for adults with acute myeloid leukemia (AML) that has come back or not responded to treatment and has a specific change in a gene called NPM1. It works by blocking a protein called menin that certain leukemia cells depend on, which can help some patients achieve remission and, in some cases, decrease their need for blood transfusions. Komzifti is taken once daily and has important side effects and monitoring requirements, so patients should review the medication guide and talk closely with their leukemia team about whether it is appropriate for them.

Reference

https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-ziftomenib-relapsed-or-refractory-acute-myeloid-leukemia-npm1-mutation?utm_medium=email&utm_source=govdelivery

Wang ES, Issa GC, Erba HP, et al. Ziftomenib in relapsed or refractory acute myeloid leukaemia (KOMET-001): a multicentre, open-label, multi-cohort, phase 1 trial. Lancet Oncol. 2024 Oct;25(10):1310-1324. doi: 10.1016/S1470-2045(24)00386-3. Erratum in: Lancet Oncol. 2024 Nov;25(11):e542. 

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