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FDA Approves Revumenib for Relapsed or Refractory AML with NPM1 Mutation
On October 24, 2025, the U.S. Food and Drug Administration (FDA) approved revumenib (Revuforj, Syndax Pharmaceuticals) for the treatment of relapsed or refractory acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. This approval applies to adults and pediatric patients one year and older who have no satisfactory alternative treatment options.
Revumenib is the first menin inhibitor approved for this AML subtype. Previously, revumenib was approved for relapsed/refractory AML with a KMT2A translocation
Menin is a protein that interacts with genes involved in leukemia cell growth. By blocking menin, revumenib aims to restore normal cell development in NPM1-mutated AML.
The study that led to revumenib approval
The FDA’s approval was based on results from the AUGMENT-101 trial (NCT04065399). This study evaluated revumenib in patients with relapsed or refractory AML confirmed to have an NPM1 mutation.
The study showed a complete response rate of 23.1%. The median duration of response was 4.5 months. Among patients who required red blood cell or platelet transfusions at the start of the trial, 17% became transfusion-independent for at least 56 days during treatment.
How is revumenib given?
Revumenib is an oral therapy, which may make treatment more accessible for patients compared to intravenous options.
The recommended dosage of revumenib varies depending on patient weight and whether the patient is also taking strong CYP3A4 inhibitors (a type of medication that affects drug metabolism). Detailed dosage information is available in the official prescribing guide.
The full prescribing information, including common side effects, for revumenib will be made available through Drugs@FDA, allowing clinicians and patients to review all safety and efficacy details.
What this approval means for patients
The approval of revumenib marks a meaningful development for patients with relapsed or refractory NPM1-mutated AML, a population with limited treatment options. This targeted approach may help some patients achieve remission and reduce transfusion needs.
As with all treatments, patients should discuss the potential benefits, risks, and monitoring requirements of revumenib with their healthcare team.
To learn more about AML and ongoing treatment developments, visit HealthTree News for AML. Staying informed empowers patients and caregivers to actively participate in care decisions.
Source:
On October 24, 2025, the U.S. Food and Drug Administration (FDA) approved revumenib (Revuforj, Syndax Pharmaceuticals) for the treatment of relapsed or refractory acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation. This approval applies to adults and pediatric patients one year and older who have no satisfactory alternative treatment options.
Revumenib is the first menin inhibitor approved for this AML subtype. Previously, revumenib was approved for relapsed/refractory AML with a KMT2A translocation
Menin is a protein that interacts with genes involved in leukemia cell growth. By blocking menin, revumenib aims to restore normal cell development in NPM1-mutated AML.
The study that led to revumenib approval
The FDA’s approval was based on results from the AUGMENT-101 trial (NCT04065399). This study evaluated revumenib in patients with relapsed or refractory AML confirmed to have an NPM1 mutation.
The study showed a complete response rate of 23.1%. The median duration of response was 4.5 months. Among patients who required red blood cell or platelet transfusions at the start of the trial, 17% became transfusion-independent for at least 56 days during treatment.
How is revumenib given?
Revumenib is an oral therapy, which may make treatment more accessible for patients compared to intravenous options.
The recommended dosage of revumenib varies depending on patient weight and whether the patient is also taking strong CYP3A4 inhibitors (a type of medication that affects drug metabolism). Detailed dosage information is available in the official prescribing guide.
The full prescribing information, including common side effects, for revumenib will be made available through Drugs@FDA, allowing clinicians and patients to review all safety and efficacy details.
What this approval means for patients
The approval of revumenib marks a meaningful development for patients with relapsed or refractory NPM1-mutated AML, a population with limited treatment options. This targeted approach may help some patients achieve remission and reduce transfusion needs.
As with all treatments, patients should discuss the potential benefits, risks, and monitoring requirements of revumenib with their healthcare team.
To learn more about AML and ongoing treatment developments, visit HealthTree News for AML. Staying informed empowers patients and caregivers to actively participate in care decisions.
Source:
about the author
Jimena Vicencio
Jimena is an International Medical Graduate and a member of the HealthTree Writing team. Currently pursuing a bachelor's degree in journalism, she combines her medical background with a storyteller’s heart to make complex healthcare topics accessible to everyone. Driven by a deep belief that understanding health is a universal right, she is committed to translating scientific and medical knowledge into clear, compassionate language that empowers individuals to take control of their well-being.
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