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  6. FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation
  1. Resources for Information | Approved Drugs

FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation

On November 15, 2024, the Food and Drug Administration approved revumenib (Revuforj, Syndax Pharmaceuticals, Inc.), a menin inhibitor, for relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older.

Full prescribing information for Revuforj will be posted on Drugs@FDA.

Efficacy and Safety

Efficacy was evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700, NCT04065399; AUGMENT-101) in 104 adult and pediatric patients (at least 30 days old) with relapsed or refractory (R/R) acute leukemia with a KMT2A translocation. Patients with an 11q23 partial tandem duplication were excluded. Revumenib was administered until disease progression, unacceptable toxicity, failure to achieve morphological leukemia-free state by 4 cycles of treatment, or hematopoietic stem cell transplantation (HSCT).

The main efficacy outcome measures were complete remission (CR) plus CR with partial hematologic recovery (CRh), the duration of CR+CRh, and conversion from transfusion dependence to independence. The CR+CRh rate was 21.2% (95% CI: 13.8, 30.3), and the median CR+CRh duration was 6.4 months (95% CI: 2.7, not estimable). Of the 22 patients achieving CR or CRh, the median time to CR or CRh was 1.9 months (range: 0.9, 5.6 months). Among the 83 patients dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 12 (14%) became independent of RBC and platelet transfusions during any 56-day post-baseline period. Of the 21 patients independent of both RBC and platelet transfusions at baseline, 10 (48%) remained transfusion independent during any 56-day post-baseline period.

The most common adverse reactions (≥20%) were hemorrhage, nausea, increased phosphate, musculoskeletal pain, infection, increased aspartate aminotransferase, febrile neutropenia, increased alanine aminotransferase, increased intact parathyroid hormone, bacterial infection, diarrhea, differentiation syndrome, electrocardiogram QT prolonged, decreased phosphate, increased triglycerides, decreased potassium, decreased appetite, constipation, edema, viral infection, fatigue, and increased alkaline phosphatase.

The recommended revumenib dose varies by patient weight and concomitant use of strong CYP3A4 inhibitors. See the prescribing information for specific dosage information. Due to an anticipated delay in commercial availability of the lowest dose strength of revumenib, which may be used to treat patients who weigh < 40 kg, revumenib will be available through an expanded access program to allow for dosing of patients who weigh < 40 kg (information available here: NCT05918913).

This review used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application 6 weeks ahead of the FDA goal date.

Expedited Programs

This application was granted priority review. Revumenib has breakthrough designation and orphan drug designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

Follow the Oncology Center of Excellence on X: @FDAOncology.

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