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| Trade names | Revuforj |
| Other names | SNDX-5613 |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a624077 |
| License data | |
| Routes of administration | By mouth |
| Drug class | Antineoplastic;menin inhibitor |
| ATC code |
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| Legal status | |
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| CAS Number | |
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| DrugBank | |
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| KEGG | |
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| PDB ligand | |
| Chemical and physical data | |
| Formula | C32H47FN6O4S |
| Molar mass | 630.82 g·mol−1 |
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Revumenib, sold under the brand nameRevuforj, is ananti-cancer medication used for the treatment ofacute leukemias harboringlysine methyltransferase 2A gene (KMT2A) rearrangements.[1] It is designed to disrupt the interaction betweenmenin andKMT2A (also known as MLL), which plays a role in thepathogenesis of these leukemias.[2] It is takenby mouth.[1]
The most common adverse reactions include 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.[3]
Revumenib was approved for medical use in the United States in November 2024.[1][3] The USFood and Drug Administration (FDA) considers it to be afirst-in-class medication.[4]
Revumenib isindicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation.[1][3]
In October 2025, the indication for revumenib was expanded for the treatment of relapsed or refractory acute myeloid leukemia with a susceptiblenucleophosmin 1 (NPM1) mutation in people who have no satisfactory alternative treatment options.[5]
The USprescribing information includes warnings and precautions fordifferentiation syndrome,QTc interval prolongation,Torsades de Pointes, andembryo-fetal toxicity.[5]
The most common adverse reactions include 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.[3]
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 participants (at least 30 days old) with relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene translocation.[3] Participants with an 11q23 partial tandem duplication were excluded.[3] Revumenib was administered until disease progression, unacceptable toxicity, failure to achieve morphological leukemia-free state by four cycles of treatment, or hematopoietic stem cell transplantation.[3]
The USFood and Drug Administration (FDA) granted the application for revumenibpriority review,breakthrough therapy, andorphan drug designations.[3]
Efficacy was evaluated in a single-arm cohort of an open-label, multi-center trial (SNDX-5613-0700, NCT04065399; AUGMENT-101).[5] A susceptible mutation was confirmed in enrolled participants using next generation sequencing or polymerase chain reaction of the last exon of NPM1.[5]
Revumenib was approved for medical use in the United States in November 2024.[3][6][7]
Revumenib is theinternational nonproprietary name.[8]
It is sold under the brand name Revuforj.[1][3]
This article incorporates text from this source, which is in thepublic domain. This article incorporates text from this source, which is in thepublic domain.