FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation
Regulatory approval: On Nov 15, 2024, the FDA approved revumenib (Revuforj; Syndax), a menin inhibitor, for adult and pediatric patients aged ≥1 year with relapsed or refractory acute leukemia with a KMT2A translocation. Approval was supported by a single-arm cohort of the open-label AUGMENT-101 trial (NCT04065399) in 104 patients. The CR+CRh rate was 21.2% with a median duration of 6.4 months (upper CI not estimable).
FDA approval (Nov 15, 2024)
The US FDA approved revumenib (Revuforj; Syndax Pharmaceuticals), a menin inhibitor, for relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients aged ≥1 year.
Evidence supporting approval
Efficacy and safety were evaluated in a single-arm cohort of an open-label, multicenter trial (SNDX-5613-0700 / AUGMENT-101; NCT04065399) enrolling 104 adult and pediatric patients (≥30 days old) with R/R acute leukemia with a KMT2A translocation; patients with 11q23 partial tandem duplication were excluded. Revumenib was given until progression, unacceptable toxicity, failure to achieve morphological leukemia-free state by 4 cycles, or HSCT.
Primary efficacy measures: CR+CRh rate, duration of CR+CRh, and transfusion dependence-to-independence conversion.
- CR+CRh: 21.2% (95% CI, 13.8–30.3)
- Median CR+CRh duration: 6.4 months (95% CI, 2.7–not estimable)
- Median time to CR/CRh (n=22): 1.9 months (range, 0.9–5.6)
- Among 83 baseline transfusion-dependent patients, 12 (14%) became independent of RBC and platelet transfusions during any 56-day post-baseline period.
Safety (selected)
Most common adverse reactions (≥20%) included hemorrhage, nausea, infection, febrile neutropenia, differentiation syndrome, QT prolongation, and multiple lab abnormalities.
Regulatory notes
The review used RTOR and an Assessment Aid, with priority review; revumenib has breakthrough and orphan drug designations.