Cancer network backs Syndax Revuforj for NPM1-mutated AML
Inclusion in NCCN guidelines is 'major milestone' as FDA weighs approval
The National Comprehensive Cancer Network (NCCN), a nonprofit alliance of 33 cancer centers, included Revuforj (revumenib) in its clinical practice guidelines for treatment of relapsed or refractory acute myeloid leukemia (AML) associated with NPM1 gene mutations, according to the therapy’s maker, Syndax.
“Given the pivotal role NCCN Guidelines play in guiding the decision-making process for clinicians, payers, patients, and other key stakeholders in the U.S. and beyond, this is a major milestone for Syndax and the entire acute leukemia community,” Nick Botwood, head of research and development and chief medical officer at Syndax, said in a company press release.
The U.S. Food and Drug Administration (FDA) is reviewing Revuforj for potential approval in the same indication, with a decision expected Oct. 25.
Clinical data show ‘strength’
Both the NCCN’s updated guidance and Syndax’s application seeking approval of Revuforj in relapsed/refractory NPM1-mutated AML are based on data from the Phase 1/2 AUGMENT-101 clinical trial (NCT04065399). Data published earlier this year showed that of 64 adults with NPM1-mutated AML who were treated with Revuforj, nearly half had a clinically meaningful response to the therapy.
“The inclusion of [Revuforj] as a recommended treatment option for [relapsed/refractory] NPM1 mutated AML in the NCCN Guidelines underscores the strength of our clinical data in this population and further solidifies revumenib’s leading position,” Botwood said.
AML is a form of blood cancer caused by the uncontrolled growth of immature blood cells. The NPM1 gene is mutated in more than one in four newly diagnosed adult AML patients. Mutations in this gene are thought to lead to abnormalities in the interactions between two proteins, KMT2A and menin, which result in abnormal genetic activity that helps to drive the abnormal growth of cancer cells.
Revuforj is an oral therapy that aims to slow AML disease progression by blocking interactions between the KMT2A and menin proteins. The therapy is FDA approved, and NCCN backed, to treat certain people with leukemia caused by mutations in the gene that provides instructions to make the KMT2A protein.
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