Revumenib was approved for adults and pediatric patients 1 year and older with relapsed or refractory acute myeloid leukemia.
The FDA has approved revumenib (Revuforj; Syndax Pharmaceuticals) for adult and pediatric patients aged 1 year and older with relapsed or refractory (RR) acute myeloid leukemia (AML) harboring a susceptible nucleophosmin 1 (NPM1) mutation, according to a news release from the agency.1
Revumenib’s efficacy and safety was affirmed in a single-arm cohort of the open-label, multicenter, dose-escalation, phase 1/2 AUGMENT-101 clinical trial (NCT04065399). In patients with RR AML and a susceptible mutation—confirmed using next generation sequencing or polymerase chain reaction—treated with revumenib, there was a 23.1% rate (95% CI, 13.5—25.2) of complete remission (CR) with partial hematological recovery (CRh).1,2
The median duration of CR and CRh in treated patients was 4.5 months (95% CI, 1.2—8.1), a significant period of efficacy. For the 46 patients at baseline who reported dependence on red blood cell (RBC) and/or platelet transfusions, 8 (17%) became independent of such transfusions during any 56-day post-baseline period, according to the study authors.1
Trial Name: A Study of Revumenib in R/R Leukemias Including Those With an MLL/KMT2A Gene Rearrangement or NPM1 Mutation (AUGMENT-101)
ClinicalTrials.gov ID: NCT04065399
Sponsor: Syndax Pharmaceuticals
Estimated Completion Date: December 15, 2027
FDA regulators recommend varying revumenib dosage dependent on a patient’s weight and concomitant use of strong CYP3A4 inhibitors. These drugs block the activity of CYP3A4, but when administered concomitantly with drugs like revumenib, it can cause toxic levels of buildup and severe side effects. Pharmacist monitoring of drug-drug interactions in this vein is critical.1,3
This is not the first regulatory action for revumenib. In November 2024, the FDA approved the treatment specifically for patients with AML harboring a lysine methyltransferase 2A gene (KMT2A) translocation. Additionally, the application for revumenib’s currently approval was granted priority review, and the candiate was previously given fast track designation.1,4
Further results from AUGMENT-101’s registration-enabling portion bolster revumenib’s efficacy in patients with AML with either NPM1 or KMT2Ar mutations. In patients evaluable for efficacy (n = 57), CR + CRh rate was 22.8% (95% CI, 12.7—35.8), which exceeded the null hypothesis of 10% (P = .0036). The overall response rate was 63.2% (95% CI, 49.3—75.6), with 15 of 22 patients (68.2%) having no detectable residual disease.1,5
Safety was also positive among this population. Grade 3 adverse events were reported and included febrile neutropenia (37.2%), differentiation syndrome (16.0%), and QTc prolongation (13.8%).1,5
Assessment of the prescribing information for revumenib for specific dosage instructions will be essential for pharmacists to complete prior to administration. Patients should be counseled on what to expect during treatment with revumenib, which is an oral, small molecule inhibitor. A patient’s medical and prescription history should be evaluated, with a pharmacist ensuring that there are no potential drug-drug interactions.1,6
Patients should ensure they convey concerns to pharmacists, including a history of adverse reactions to other small molecule inhibitors. If a patient has a medical history of heart failure, heart rhythm problems—also known as long QT syndrome—or hypokalemia, use of revumenib could worsen these conditions. Pharmacists, along with the entire health care team, should judiciously monitor patients being treated with revumenib throughout their course and following the completion of their treatment.1,6
Stay informed on drug updates, treatment guidelines, and pharmacy practice trends—subscribe to Pharmacy Times for weekly clinical insights.
