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Revumenib (SNDX-5613), an oral menin inhibitor, is FDA-approved (Nov 2024) for relapsed/r...

Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.

IDBMA-KMT2A-REARR-AML
TypeActionability
Statusreviewed 2026-05-03 | pending_clinical_signoff | actionability review required
DiseasesDIS-AML
SourcesSRC-ELN-AML-2022 SRC-NCCN-AML-2025

Actionability Facts

BiomarkerBIO-KMT2A-REARRANGEMENT
VariantKMT2A (MLL) rearrangement — any fusion partner (KMT2A-MLLT3/AF9, KMT2A-MLLT4/AF6, KMT2A-ELL, KMT2A-MLLT1/ENL, KMT2A-AF1Q, KMT2A-AFDN among others; detected by FISH, RNA-seq, or cytogenetics)
DiseaseDIS-AML
ESCAT tierIA
Recommended combinationsrevumenib 163 mg PO BID (with strong CYP3A4 inhibitor: 95 mg BID) monotherapy — R/R KMT2A-rearranged AML post ≥2 prior lines, revumenib + venetoclax + azacitidine (investigational; phase II enrollment ongoing as of 2026)
Evidence summaryRevumenib (SNDX-5613), an oral menin inhibitor, is FDA-approved (Nov 2024) for relapsed/refractory KMT2A-rearranged acute leukemia (AML and ALL). AUGMENT-101 (Issa et al. NEJM 2023; Erba et al. ASH 2023 update): KMT2A-rearranged R/R AML/ALL — ORR 23% (CR + CRh + CRi), complete remission with full recovery 18%; mOS 8.0 mo. FDA accelerated approval based on remission rate as surrogate endpoint. KMT2A rearrangement is present in ~10% of adult AML (more common in secondary/therapy-related AML and infant leukemia). Menin is an essential co-factor of KMT2A-fusion oncoproteins — revumenib disrupts menin-MLL interaction, restoring differentiation block. Combination strategies under evaluation: revumenib + venetoclax + azacitidine (phase II ongoing). ELN 2022 risk: KMT2A-rearranged AML classified as intermediate risk (specific partner-dependent — MLLT3 intermediate; others may be adverse).

Notes

ESCAT IA (accelerated approval; confirmatory phase III AUGMENT-101P ongoing). Drug-drug interaction caution: revumenib is a CYP3A4 substrate; strong CYP3A4 inhibitors (azoles) require dose reduction to 95 mg BID. QTc prolongation monitoring required (baseline ECG, potassium/magnesium correction). Differentiation syndrome risk (similar to ATRA in APL) — monitor for fever, hypotension, pulmonary infiltrates; prophylactic corticosteroids under evaluation. Bridge to alloHCT should be planned for all eligible patients achieving remission. NPM1-mutated AML is a separate FDA-approved indication for revumenib (same drug, different biomarker mechanism) — see BMA for NPM1 when created.

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