The FDA grants precedence evaluation to Revuforj’s supplemental new drug utility to deal with R/R mNPM1 AML: © inventory.adobe.com.
The U.S. Meals and Drug Administration (FDA) has granted precedence evaluation to the supplemental new drug utility for Revuforj (revumenib) for the therapy of relapsed or refractory mutant NPM1 acute myeloid leukemia (AML), in response to a information launch from Syndax.
Revuforj is an oral, first-in-class menin inhibitor used to deal with sure sorts of relapsed or refractory leukemia with particular genetic adjustments.
The supplemental new drug utility is being reviewed by the FDA’s real-time oncology evaluation program, which permits for a extra environment friendly evaluation course of and shut engagement between the company and the sponsor all through the submission. The applying has been assigned a goal motion date of October 25, 2025, underneath the Prescription Drug Consumer Payment Act.
“We’re happy that the FDA has granted precedence evaluation to our supplemental new drug utility in relapsed or refractory mutant NPM1 AML, a submitting which builds on the preliminary approval of Revuforj for relapsed or refractory acute leukemia with a KMT2A translocation in 2024,” Michael A. Metzger, chief govt officer, mentioned within the information launch. “Syndax is uniquely positioned to proceed main this thrilling new therapeutic class with a first- and best-in-class menin inhibitor supported by compelling pivotal information throughout the broadest inhabitants of sufferers and a robust basis already established amongst clinicians, payers, and different key stakeholders.”
Based on the U.S. FDA web site, a precedence evaluation designation for a supplemental new drug utility is supposed to speed up the analysis of the drug, with an estimated resolution timeline of six months in contrast with the same old 10. Furthermore, this course of is particularly reserved for supplemental new drug functions that may doubtlessly characterize a significant development for the therapy of a critical situation in contrast with present choices, if accredited.
This potential label enlargement is backed by outcomes from the AUGMENT-101 trial, which studied Revuforj on this inhabitants. Findings had been printed in Blood in Could 2025 and shared on the European Hematology Affiliation’s annual assembly in June 2025.
Revuforj is an oral therapy that blocks menin and was first accredited by the FDA in 2024 for adults and youngsters a minimum of one 12 months outdated with relapsed or refractory acute leukemia pushed by a KMT2A translocation.
Amongst sufferers within the AUGMENT-101 trial, 21.2% achieved full remission plus full remission with partial hematological restoration with a median period of 6.4 months. As well as, of the 22 sufferers who achieved both full response or full remission with partial hematological restoration, the median time to both of these outcomes was 1.9 months.
The commonest uncomfortable side effects, which occurred in a minimum of 20% of sufferers, included nausea, bleeding, musculoskeletal ache, excessive phosphate ranges, elevated aspartate aminotransferase, an infection, febrile neutropenia, elevated intact parathyroid hormone, elevated alanine aminotransferase, diarrhea, bacterial an infection, QT prolongation, differentiation syndrome, elevated triglycerides, decreased phosphate, decreased potassium, constipation, decreased urge for food, fatigue, viral an infection and elevated alkaline phosphatase.
The brand new utility now underneath evaluation goals to increase its use to sufferers with relapsed or refractory AML who carry an NPM1 mutation, the commonest genetic alteration on this illness.
Extra Data About Revuforj and Mutant NPM1 AML
Mutations within the NPM1 gene, seen in about 30% of adults with AML, outline a subtype generally known as mutant NPM1 AML — an aggressive type of the illness with excessive relapse charges and restricted therapy choices, in response to the discharge. These leukemias are strongly depending on the menin-KMT2A interplay, which helps drive most cancers development by activating particular genes. Disrupting this interplay has proven promise in lowering disease-driving indicators. Whereas mutant NPM1 AML could be recognized by customary diagnostic strategies, no accredited remedies at present goal its underlying biology, leaving a major unmet want for sufferers with relapsed or treatment-resistant illness.
Further research of Revuforj, together with in newly recognized sufferers, are underway or deliberate, combining the drug with customary remedies in NPM1-mutated or KMT2A-rearranged leukemia. Revuforj has obtained a number of FDA and European designations, together with orphan drug, quick monitor, and breakthrough remedy designations.
Reference
“FDA grants precedence evaluation for supplemental utility of Revuforj in relapsed or refractory mutant NPM1 acute myeloid leukemia” Information launch. Syndax Prescription drugs, Inc. June 24, 2025.
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