In a development that could reshape treatment for a difficult-to-treat subtype of acute myeloid leukemia (AML), the U.S. Food and Drug Administration has granted priority review to the supplemental new drug application for revumenib in patients with relapsed or refractory AML carrying the NPM1 mutation. This decision reflects both promising clinical data and pressing unmet need in this patient population.

Revumenib (also known by its brand name Revuforj) is a first-in-class menin inhibitor. It had previously been approved for AML cases with KMT2A rearrangements, in adults and children. The new application seeks approval for a broader set of patients – those whose disease still persists or returns after treatment, and who have the NPM1 mutation.

The application is supported by data from the AUGMENT-101 trial, a Phase 1/2 clinical study. In that trial, patients with NPM1-mutant, relapsed/refractory AML showed responses that convinced the FDA to consider this expansion. The reviewing process has been expedited under the Real-Time Oncology Review program. The goal under priority review is to shave time off the traditional review period so that patients may access the therapy sooner if approved.

For patients, this could matter a lot. AML with an NPM1 mutation is relatively common among the genetic subtypes of AML. While patients with NPM1 mutant disease sometimes respond well to initial treatment, relapse and refractoriness remain serious obstacles. Existing therapies often have limited effectiveness once standard care fails. A treatment like revumenib, especially one that has shown clear activity in early trials, offers a chance to fill a gap.

Of course, there are caveats. While the trial data are promising, longer-term outcomes (duration of response, survival) and safety in larger populations remain to be fully characterised. Side effects typical of menin inhibitors and other novel small molecules will need close surveillance once the therapy is more broadly used. Also cost, access, and how revumenib will fit into evolving AML treatment algorithms are open questions.

Regulatory timing is now in focus. With the priority review in place regulatory guidance suggests a target action date will be set. If everything goes smoothly, revumenib could become an approved option for NPM1-mutant, relapsed or refractory AML by later in 2025 or early 2026. For hematologists and patients, that timeline is being watched closely.

This news underscores a broader trend: genetic subtyping of AML and other cancers is increasingly central to development decisions. As therapies become more specific, it becomes critical to move quickly, but also to maintain rigorous evidence of benefit and safety. Revumenib’s priority review is a reminder that regulators and researchers are aligned in pushing forward for patients with few good options.